Curriculum vitae
PERSONAL INFORMATION
Annemieke Aartsma-Rus
WORK EXPERIENCE January 2000–June 2000
Technician Biomedical Primate Research Center, (Netherlands) Cell and protein studies for multiple sclerosis models
July 2000–August 2004
PhD Student Leiden University, (Netherlands) PhD research on the topic "Antisense-mediated exon skipping for Duchenne muscular dystrophy" under supervision of Judith van Deutekom and Gert-Jan van Ommen
September 2004–September 2007
Postdoc Leiden University Medical Center, (Netherlands) Optimization of antisense-mediated exon skipping
December 2007–February 2010
Assistant Professor Leiden University Medial Center, (Netherlands) Group leader of exon skipping group
March 2010–October 2015
Leiden University Medical Center Associate Professor, (Netherlands) Group leader on antisense mediated exon skipping for Duchenne muscular dystrophy and other rare diseases
January 2012–Present
Visiting Professor Newcastle University, (United Kingdom)
October 2015–Present
Professor Leiden University Medical Center, (Netherlands) Professor of Translational Genetics
EDUCATION AND TRAINING September 1995–August 1996
Bachelor of Science Leiden University, (Netherlands) Biomedical Sciences
September 1996–January 2000
Master of Science Leiden University, (Netherlands) Biomedical Sciences, major in genetics
July 2000–January 2005
PhD Leiden University, (Netherlands)
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PhD research on antisense-mediated exon skipping for Duchenne muscular dystrophy ADDITIONAL INFORMATION Expertise
Duchenne muscular dystrophy Exon skipping Antisense oligonucleotides Dystrophin Animal models Rare diseases Therapy development Serum biomarkers Splicing manipulation Dystrophin quantification Duchenne pathology
Publications
1. Pulyakhina I, Gazzoli I, 't Hoen PA, Verwey N, den Dunnen JT, Aartsma-Rus A, Laros JF. SplicePie: a novel analytical approach for the detection of alternative, non-sequential and recursive splicing. Nucleic Acids Res. 2015 Oct 7. 2. Switzar L, Nicolardi S, Rutten JW, Oberstein SA, Aartsma-Rus A, van der Burgt YE. In-Depth Characterization of Protein Disulfide Bonds by Online Liquid Chromatography-Electrochemistry-Mass Spectrometry. J Am Soc Mass Spectrom. 2015 Sep 14. 3. Kinnett K, Rodger S, Vroom E, Furlong P, Aartsma-Rus A and Bushby K: Imperatives for DUCHENNE MD: a Simplified Guide to Comprehensive Care for Duchenne Muscular Dystrophy. PLoS Curr. 2015 epub Aug 7 4. Jirka S and Aartsma-Rus A. An update on RNA-targeting therapies for neuromuscular disorders. Curr Opin Neurol. 2015 epub Aug 14. 5. Guiraud S, Aartsma-Rus A, Vieira NM, Davies KE, van Ommen GJ, Kunkel LM. The Pathogenesis and Therapy of Muscular Dystrophies. Annu Rev Genomics Hum Genet. 2015 Jun 4. (Epub) 6. Van den Bergen J, Aartsma-Rus A. Response to: Studying the role of dystrophin-associated proteins in influencing Becker muscular dystrophy disease severity. Neuromuscul Disord. 2015 Jun;25(6):530-1. 7. ramlich M, Pane LS, Zhou Q, Chen Z, Murgia M, Schötterl S, Goedel A, Metzger K, Brade T, Parrotta E, Schaller M, Gerull B, Thierfelder L, Aartsma-Rus A, Labeit S, Atherton JJ, McGaughran J, Harvey RP, Sinnecker D, Mann M, Laugwitz KL, Gawaz MP, Moretti A: Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy. EMBO Mol Med. 2015 epub Mar 9. 8. van den Bergen JC, Wokke BH, Hulsker MA, Verschuuren JJ, Aartsma-Rus A: Studying the role of dystrophin-associated proteins in influencing Becker muscular dystrophy disease severity. Neuromuscul Disord. 2015;25(3):231-7. 9. Bladen CL, Salgado D, Monges S, Foncuberta ME, Kekou K, Kosma K, Dawkins H, Lamont L…, Aartsma-Rus A, Beroud C and Lochmüller H. The TREAT-NMD DMD Global database: Analysis of More Than 7000 Duchenne Muscular Dystrophy Mutations. Hum Mutat. 2015 epub Jan 21 10. Arechavala-Gomeza V, Khoo B, Aartsma-Rus A. Splicing modulation therapy in the treatment of genetic diseases. Appl Clin Genet. 2014 ;7:245-52. 11. van den Bergen JC, Hiller M, Böhringer S, Vijfhuizen L, Ginjaar HB, Chaouch A, Bushby K, Straub V, Scoto M, Cirak S, Humbertclaude V, Claustres M, Scotton C, Passarelli C, Lochmüller H, Muntoni F, Tuffery-Giraud S, Ferlini A, Aartsma-Rus A, Verschuuren JJ, 't Hoen PA, Spitali P: Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants. J Neurol Neurosurg Psychiatry. 2014 Dec 4 epub 12. van Putten M, Young C, van den Berg S, Pronk A, Hulsker M, Karnaoukh TG, Vermue R, van Dijk KW, de Kimpe S and Aartsma-Rus A: Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2014; 3:e211.
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13. Lynn S, Aartsma-Rus A, Bushby K, Furlong P, Goemans N, De Luca A, Mayhew A, McDonald C, Mercuri E, Muntoni F, Pohlschmidt M, Verschuuren J, Voit T, Vroom E, Wells DJ, Straub V: Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy. Neuromuscul Disord. 2014 epub Sept 11. 14. Vojinovic D, Adams HH, van der Lee SJ, Ibrahim-Verbaas CA, Brouwer R, van den Hout MC, Oole E, van Rooij J, Uitterlinden A, Hofman A, van IJcken WF, Aartsma-Rus A, van Ommen GB, Ikram MA, van Duijn CM and Amin N. The dystrophin gene and cognitive function in the general population. Eur J Hum Genet. 2014 (epub Sep 17). 15. Aartsma-Rus A, Ferlini A, Goemans N, Pasmooij AM, Wells DJ Dr, Bushby K, Vroom E, Balabanov P: Translational and regulatory challenges for exon skipping therapies. Statement paper based on a BM1207 COST Action meeting involving patient representatives, clinicians, preclinical scientists and regulators. Hum Gene Ther. 2014 (epub Sep 3). 16. Aartsma-Rus A, Ferlini A, Vroom E: Biomarkers and surrogate endpoints in Duchenne: Meeting report. Neuromuscul Disord. 2014;24:743-5. 17. Aartsma-Rus A: Dystrophin quantification in clinical trials. JND. 2014; 1: 41-53. 18. Ayoglu B, Chaouch A, Lochmüller H, Politano L, Bertini E, Spitali P, Hiller M, Niks E, Gualandi F, Pontén F, Bushby K, Aartsma-Rus A, Schwartz E, Le Priol Y, Straub V, Uhlén M, Cirak S, 't Hoen PA, Muntoni F, Ferlini A, Schwenk JM, Nilsson P, Al-Khalili Szigyarto C. Affinity proteomics within rare diseases: a BIO-NMD study for blood biomarkers of muscular dystrophies. EMBO Mol Med. 2014 epub Jun 11 19. Aartsma-Rus A and van Putten M: Assessing functional performance in the mdx mouse model. J Vis Exp. 2014, epub Mar 27 (85) 20. Kemaladewi DU, Pasteuning S, van der Meulen JW, van Heiningen SH, van Ommen GJ, Ten Dijke P, Aartsma-Rus A, 't Hoen PA, Hoogaars WM: Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor. Mol Ther Nucleic Acids. 2014; 3:e156. 21. Wokke BH, van den Bergen JC, Versluis MJ, Niks EH, Milles J, Webb AG, van Zwet EW, Aartsma-Rus A, Verschuuren JJ and Kan HE: Quantitative MRI and strength measurements in the assessment of muscle quality in Duchenne muscular dystrophy. Neuromuscul Disord. 2014; 24: 40916. 22. Tremblay JP, Aartsma-Rus A, Bogdanove A, Ferreira MB, Bueren J and Huard J: Development of a web course on gene therapy by the international consortium of gene therapy. Mol Ther. 2014; 22: 482. 23. Verhaart IE, van Vliet-van den Dool L, Sipkens JA, de Kimpe SJ, Kolfschoten IG, van Deutekom JC, Liefaard L, Ridings JE, Hood SR and Aartsma-Rus A: The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice. Mol Ther Nucleic Acids. 2014; 18: e148. 24. Aartsma-Rus A: Antisense-mediated exon skipping: networking to meet opportunities and to overcome challenges. Nucleic Acid Ther 2014; 24:1-3. 25. Veltrop M and Aartsma-Rus A. Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseases. Exp Cell Res. 2014; 325: 50-55. 26. Raz V, Buijze H, Raz Y, Verwey N, Anvar SY, Aartsma-Rus A and van der Maarel SM: A Novel Feed-Forward Loop between ARIH2 E3-Ligase and PABPN1 Regulates Aging-Associated Muscle Degeneration. Am J Pathol. 2014; 184: 1119-31. 27. van Putten M, van der Pijl EM, Hulsker M, Verhaart IE, Nadarajah VD, van der Weerd L and Aartsma-Rus A: Low dystrophin levels in heart can delay heart failure in mdx mice. J Mol Cell Cardiol. 2014; 69: 17-23. 28. Martin FC, Hiller M, Spitali P, Oonk S, Dalebout H, Palmblad M, Chaouch A, Guglieri M, Straub V, Lochmüller H, Niks EH, Verschuuren JJ, Aartsma-Rus A, Deelder AM, van der Burgt YE, 't Hoen PA: Fibronectin is a serum biomarker for Duchenne muscular dystrophy. Proteomics Clin Appl. 2014; 8: 269-78 29. Karkampouna S, Kruithof BP, Kloen P, Obdeijn MC, van der Laan AM, Tanke HJ, Kemaladewi DU, Hoogaars WM, 't Hoen PA, Aartsma-Rus A, Clark IM, Ten Dijke P, Goumans MJ, Kruithof-de Julio M: Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides. Mol Ther Nucleic Acids. 2014; 3: e142 30. Jirka SM, Heemskerk H, Tanganyika-de Winter CL, Muilwijk D, Pang KH, de Visser PC, Janson A, Karnaoukh TG, Vermue R, 't Hoen PA, van Deutekom JC, Aguilera B and Aartsma-Rus A: Peptide Conjugation of 2'-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake
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and Exon Skipping in mdx Mice. Nucleic Acid Ther 2014; 24: 25-36. 31. Evers MM, Tran HD, Zalachoras I, Meijer OC, den Dunnen JT, van Ommen GJ, Aartsma-Rus A, van Roon-Mom WM: Preventing Formation of Toxic N-Terminal Huntingtin Fragments Through Antisense Oligonucleotide-Mediated Protein Modification. Nucleic Acid Ther. 2014; 24: 4-12. 32. Van den Bergen JC, Schade van Westrum SM, Dekker L, van der Kooi AJ, Straathof CS, Aartsma-Rus A, Verschuuren JJGM and Ginjaar HB: The clinical characterization of Becker patients with various in-frame deletions predicts favorable outcome for exon skipping therapy. J Neurol Neurosurg and Psych 2014; 85: 92-8. 33. van den Bergen JC, Wokke BH, Janson AA, van Duinen SG, Hulsker MA, Ginjaar HB, van Deutekom JC, Aartsma-Rus A, Kan HE, Verschuuren JJ: Dystrophin levels and clinical severity in Becker muscular dystrophy patients. J Neurol Neurosurg Psychiatry. 2014; 85: 747-53 34. Spitali P, Grumati P, Hiller M, Chrisam M, Aartsma-Rus A, Bonaldo P: Autophagy is Impaired in the Tibialis Anterior of Dystrophin Null Mice. PLoS Curr MD. 2013 epub Nov 22, 5 35. Veltrop M, van der Kaa J, Claassens J, van Vliet L, Verbeek S and Aartsma-Rus A: Generation of embryonic stem cells and mice for Duchenne research. PLoS Curr MD 2013 Sep 10, 5 36. Spitali P, van den Bergen JC, Verhaart IEC, Wokke B, Janson AM, van den Eijnde R, den Dunnen JT, Laros JF, Verschuuren JJGM, ‘t Hoen PAC and Aartsma-Rus A: DMD transcript imbalance determines dystrophin levels. FASEB J 2013; 27: 4909-16. 37. De Brouwer AP, Nabuurs SB, Verhaart IE, Oudakker AR, Hordijk R, Yntema HG…..Aartsma-Rus A, Hamel BC, van Bokhoven H and Kleefstra T: A 3-base pair deletion, c.9711_9713del, in DMD results in intellectual disability without muscular dystrophy. Eur J Hum Genet 2014; 22: 480-5. 38. Aartsma-Rus A and Muntoni F: 194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8-10 December 2012, Naarden, the Netherlands. Neuromusc Disord 2013; 23: 934-44. 39. Shi S, Cai J, de Gorter DJJ, Sanchez Duffhues G, Kemaladewi DU, Hoogaars WM, Aartsma-Rus A, ‘t Hoen PAC, ten Dijke P: Antisense-Oligonucleotide Mediated Exon Skipping in Activin-ReceptorLike Kinase 2: Inhibiting the Receptor That Is Overactive in Fibrodysplasia Ossificans Progressiva. Plos One 2013; e69096. 40. Dick E, Kalra S, Anderson D, George V, Ritson M, Laval S, Barresi R, Aartsma-Rus A, Lochmuller H, Denning C: Exon skipping and gene transfer restore dystrophin expression in hiPSCcardiomyocytes harbouring DMD mutations. Stem Cells and Development 2013. 22: 2714-24. 41. Klymiuk N, Blutke A, Graf A, Krause S…..Aartsma-Rus A….Wolf E: Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle. Hum Mol Genet 2013; 22: 4368-82. 42. Evers MM, Tran HD, Zalachoras I, Pepers BA, Meijer OC, den Dunnen JT, van Ommen GJ, Aartsma-Rus A, van Roon-Mom WM. Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon. Neurobiol Dis. 2013; 58: 49-56. 43. Verhaart IEC, Tanganyika-de Winter CL, Karnaoukh TG, Kolfschoten IGM, de Kimpe SJ, van Deutekom JCT and Aartsma-Rus A: Dose-dependent pharmacokinetic profiles of 2’-O-methyl phosphorothioate antisense oligonucleotides in mdx mice. Nucleic Acids Therapeutics 2013; 23: 22837. 44. Van Putten M, Hulsker M, Young C, Nadarajah VD, Heemskerk H, van der Weerd L, ‘t Hoen PAC, van Ommen GJB and Aartsma-Rus A: Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double knockout mice. FASEB 2013; 27: 2484-95. 45. van Ommen GJ and Aartsma-Rus A: Advances in therapeutic RNA-targeting. N. Biotechnol. 2013 Jan 28. S1871-6784. 46. Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, et al.: Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther. 2013; 21: 266-8. 47. McCormack P, Woods S, Aartsma-Rus A, Hagger L, Herczegfalvi A, Heslop E, Irwin J, Kirschner J, Moeschen P, Muntoni F, Ouillade MC, Rahbek J, Rehmann-Sutter C, Rouault F, Sejersen T, Vroom E, Straub V, Bushby K, Ferlini A: Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: “translating the translational”. Plos Curr MD 2013, Jan 10, 5 48. Zalachoras I, Gootaers G, van Weert LT, Aubert Y, de Kreij SR, Datson NA, van Roon-Mom WM, Aartsma-Rus A and Meijer OC: Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain. BMC Neurosci. 2013; 14: 5
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49. Yilmaz-Elis S, Aartsma-Rus A, Hoen PAC, Safdar H, Breukel C, van Vlijmen BJM, van Deutekom JCT, de Kimje SJ, van Ommen GJB and Verbeek SJ: IL-1 Signaling by Antisense Oligonucleotide Mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP). Mol Ther NA 2013; 2: e66 50. Verhaart I and Aartsma-Rus A: The effect of 6-thioguanine on alternative splicing and antisensemediated exon skipping treatment for Duchenne muscular dystrophy. Plos Curr MD 2012; Dec 12, 4 51. Lemmers RJ, Tawil R, Petek LM, Balog J, Block GJ….Aartsma-Rus AM…and van der Maarel SM: Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2. Nat Genet. 2012; 44: 1370-4. 52. Tanganyika-de Winter CL, Heemskerk H, Karnaoukh TG, van Putten M, de Kimpe SJ, van Deutekom JCT and Aartsma-Rus A: Long term exon skipping studies with 2’-O-methyl phosphorothioate antisense oligonucleotides in dystrophic mouse models. Mol Ther 2012; 1:e44. 53. Verhaart IEC and Aartsma-Rus A: Gene Therapy for Duchenne Muscular Dystrophy. Curr Opin Neurol 2012; 25:288-96. 54. Yilmaz-Elis S, Aartsma-Rus A, Vroon A, van Deutekom J, de Kimpe S, 't Hoen PA, van Ommen GJ, Verbeek JS: Antisense oligonucleotide mediated exon skipping as a potential strategy for the treatment of a variety of inflammatory diseases such as rheumatoid arthritis. Ann Rheum Dis 2012; 71 Suppl 2:i75-7. 55. Spitali P and Aartsma-Rus A: Progress in splice-modulating therapies for human disease. Cell 2012; 148: 1085-8 56. van Putten M, Hulsker MH, Nadaraja VD, van Heiningen SH, van Huizen E, van Iterson M, Admiraal P, Messemaker T, den Dunnen JT, ’t Hoen PAC and Aartsma-Rus A: The effect of low levels of dystrophin on mouse muscle function and pathology. PLoS ONE 2012; 7: e31937 57. Van Putten M, Kumar D, Hulsker MH, Hoogaars WMH, Plomp JJ, van Opstal A, van Iterson M, Admiraal P, van Ommen GJB, ’t Hoen PAC and Aartsma-Rus A: Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains. Neuromuscul Dis 2012; 22: 406-17 58. Verhaart IE, van Duijn RJ, den Adel B, Roest AA, Verschuuren JJ, Aartsma-Rus A and van der Weerd L: Assessment of cardiac function in three mouse dystrophinopathies by magnetic resonance imaging. Neuromuscul Disord 2011; 22: 418-26 59. Kemaladewi DU, de Gorter DJ, Aartsma-Rus A, van Ommen GJ, Ten Dijke P, ’t Hoen PA and Hoogaars WM: Cell-type specific regulation of myostatin signalling. FASEB J 2012; 26: 1462-72 60. Verhaart IE, Heemskerk H, Karnaoukh TG, Kolfschoten IG, Vroon A, van Ommen GJ, van Deutekom JC, Aartsma-Rus A: Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy. Hum Gene Ther. 2012; 23:262-73. 61. ‘t Hoen PA, Jirka SM, Ten Broeke BR, Schultes EA, Aguilera B, Pang KH, Heemskerk H, AartsmaRus A, van Ommen GB and den Dunnen JT: Phage display screening without repetitious selection rounds. Anal Biochem 2011. 421: 622-31. 62. Anthony K, Cirak S, Torelli S, Tasca G, Feng L, Arechavala-Gomeza V, Armaroli A, Guglieri M, Straathof CS, Verschuuren JJ, Aartsma-Rus A, et al: Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials. Brain 2011; 134: 3544-56 63. Evers MM, Pepers BA, van Deutekom JCT, Mulders SA, den Dunnen JT, Aartsma-Rus A, van Ommen GJB and van Roon-Mom WCM: Targeting several CAG repeat expansion diseases by a single antisense oligonucleotide. PLoS One 2011; e24308 64. Zalachoras I, Evers MM, van Roon-Mom WCM, Aartsma-Rus A and Meijer OC: Antisensemediated RNA targeting: versatile and expedient genetic manipulation in the brain. Front Mol Neurosci 2011; 4:10 65. Nadarajah VD, van Putten M, Chaouch A, Garrood P….Aartsma-Rus A et al: Serum Matrix Metalloproteinase-9 (MMP-9) as a biomarker for monitoring Duchenne Muscular Dystrophy (DMD) disease progression. Neuromuscul Disord 2011; 21: 569-78 66. Van Putten M and Aartsma-Rus A: Opportunities and challenges for the development of antisense treatment in neuromuscular disorders. Exp Opin Biol Ther 2011; 11: 1025-37 67. Kemaladewi DU, Hoogaars WM, van Heiningen SH, Terlouw S, de Gorter DJ, den Dunnen JT, van Ommen GJB, Aartsma-Rus A, ten Dijke P and ’t Hoen PA: Dual exon skipping for myostatin and dystrophin for Duchenne muscular dystrophy. BMC Med Genomics 2011; 4: 36 68. Pichavant C, Aartsma-Rus A, Clemens PR, Davies KE, Dickson G, Takeda S, Wilton SD, Wolff JA, Wooddell CI, Xiao X and Tremblay JP: Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD. Mol Ther 2011; 19: 830-40
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69. Goemans N, Tulinius M, van den Akker JT…..Aartsma-Rus A…and van Deutekom JCT: Systemic administration of PRO051 in Duchenne’s muscular dystrophy. N Engl J Med 2011; 364: 1513-22 70. Aartsma-Rus A, on behalf of the working group: Effects of long-term treatment and combination therapeutics for neuromuscular disorders. Report of a TREAT-NMD/NIH “Bringing down the barriers – Translational medicine in inherited neuromuscular diseases” conference working group. Neuromuscul Disord 2011; 21: 151-6. 71. Aartsma-Rus A, The risks of therapeutic misconception and n=1 “trials” in rare diseases. Requests for individual “trials” of exon skipping risk setting back the entire field. Neuromuscul Disord 2011; 21: 13-15. Aartsma-Rus A, Response to a comment to this paper, Neuromuscul Disord 2011; 21: 527-8 72. Aartsma-Rus A, den Dunnen JT and van Ommen GJB: New insights in gene therapy: the example of Duchenne muscular dystrophy. Ann NY Acad Sci 2010; 1214:199-212. 73. Shi ST, Hoogaars WM, de Gorter DJ, van Heiningen SH, Lin HY, Hong CC, Kemaladewi DU, Aartsma-Rus A, Ten Dijke P and ‘t Hoen PA: BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model. Neurobiol Dis 2011; 41: 353-60 74. Aartsma-Rus A: Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy. RNA Biology 2010; 7: 4 75. Spitali P, Heemskerk JA, Vossen RHAM, Ferlini A, den Dunnen JT, ‚t Hoen PAC and Aartsma-Rus A: Accurate quantification of dystorphin mRNA and exon skipping levels in Duchenne muscular dystrophy. Lab Invest 2010; 90: 1396-402. 76. Heemskerk JA, de Winter CL, van Kuik P…Aartsma-Rus A and van Deutekom JCT: Pre-clinical PK and PD studies on 2’O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx Mouse model. Mol Ther 2010; 18: 1210-7. 77. Aartsma-Rus A, Houlleberghs H, van Deutekom JCT, van Ommen GJB and ’t Hoen PAC: Exontargeting antisense oligonucleotides outperform splice site targeting antisense oligonucleotides in the modulation of DMD splicing. Oligonucleotides 2010; 20: 69-77. 78. Muntoni F, Meeting Steering Committee and TREAT-NMD Network: The development of antisense oligonucleotides therapies for Duchenne muscular dystrophy: Report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009. Neuromuscul Disord 2010; 20: 355-62. 79. Van Putten M, de Winter CL, van Roon-Mom W, van Ommen GJB, ’t Hoen PAC and AartsmaRus A: A mild exercise regime does not affect disease parameters in the mdx mouse. Neuromuscul Disord 2010; 20: 273-80. 80. Helderman-van den Enden AT, Straathof CS, Aartsma-Rus A, den Dunnen JT, Verbist BM, Bakker E, Verschuuren JJ and Ginjaar HB: Becker muscular dystrophy patients with deletions around exon 51; A promising outlook for exon skipping therapy in Duchenne patients. Neuromuscul Disord 2010; 20: 251-4. 81. Aartsma-Rus A, Singh KH, Fokkema IF, Ginjaar IB, van Ommen GJ, Den Dunnen JT, van der Maarel SM: Therapeutic exon skipping for dysferlinopathies? Eur J Hum Genet 2010; 18: 889-94. 82. Popplewell LJ, Adkin C, Arechavala-Gomeza V, Aartsma-rus A, de Winter CL, Wilton SD, Morgan JE, Muntoni F, Graham IR, Dickson G: Comparative analysis of antisense oligonucleotides sequences targeting exon 53 of the human DMD gene; Implications for future clinical trials. Neuromuscul Disord 2010; 20: 102-110. 83. Aartsma-Rus A and van Ommen GJB: Progress in therapeutic antisense applications for neuromuscular disorders. Eur J Hum Gen 2010; 18: 146-53. 84. Heemskerk JA, de Winter CL, van Ommen GJB, van Deutekom JCT and Aartsma-Rus A: Development of antisense-mediated exon skipping as a treatment for Duchenne muscular dystrophy. Ann NY Acad Sc 2009; 1175: 71-9. 85. Aartsma-Rus, A and Van Ommen GJB: Less is more: therapeutic exon skipping for Duchenne muscular dystrophy. Lancet Neur 2009; 8: 873-5. 86. Van de Vosse E, Verhard EM, de Paus RA, ven Deutekom JCT, Aartsma-Rus A and van Dissel JT: Antisense-mediated exon skipping to correct IL12Rβ1 deficiency in T-cells. Blood 2009; 113: 454855. 87. Aartsma-Rus A, Fokkema I, Verschuuren JJ, Ginjaar HB, van Deutekom JCT, van Ommen GJB and den Dunnen JT: Applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat 2009; 30: 293-9. 88. Heemskerk JA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg G, van Ommen GJB, van Deutekom JCT and Aartsma-Rus A: In vivo comparison of 2’-O-methyl
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phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med 2009; 11: 257-66. 89. Van Vliet L, van Deutekom JCT, van Ommen GJB and Aartsma-Rus A: assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy. BMC Med Genet 2008; 9: 105. 90. Aartsma-Rus A, van Vliet L, Hirschi M, Janson AM, Heemskerk JA, de Winter CL, de Kimpe SJ, van Deutekom JCT, ’t Hoen PAC and van Ommen GJB: Guidelines for antisense oligonucleotide design and insight in splice modulating mechanisms. Mol Ther 2009; 17: 548-53. 91. Van Ommen GJB, van Deutekom JCT and Aartsma-Rus A: The therapeutic potential of antisense-mediated exon skipping. Curr Opin Mol Ther 2008; 10: 140-49. 92. Van Deutekom JCT, Janson AM, Ginjaar HB, Aartsma-Rus A et al: Local dystrophin restoration with antisense oligonucleotides PRO051. N Engl J Med 2007; 357: 2677-86. 93. Arechavala-Gomeza V, Graham IR, Popplewell LJ, Adams AM, Aartsma-Rus A, Kinali M, Morgan JE, van Deutekom JCT, Wilton SD, Dickson G and Muntoni F: Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. Hum Gene Ther 2007; 18: 798-810. 94. Aartsma-Rus A and van Ommen GJB: Antisense-mediated exon skipping: A versatile tool with research and therapeutic applications. RNA 2007; 13: 1609-24. 95. Aartsma-Rus A, Janson AAM, van Ommen GJB, van Deutekom JCT: Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy. BMC Med Genet 2007; 8: 43. 96. Aartsma-Rus A, Janson AAM, Heemskerk JA, de Winter CL, van Ommen GJB and van Deutekom JCT: Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides. Ann NY Acad Sc 2006; 1082: 74-6. 97. White SJ, Aartsma-Rus A, Flanigan KM, Weiss RB, Kneppers ALJ, Lalic T, Janson AAM, Ginjaar HB, Breuning MH and den Dunnen JT: Duplications in the DMD gene. Hum Mutat 2006; 27: 938-45. 98. ‘t Hoen PAC, van der Wees CGC, Aartsma-Rus A, Turk R, Goyenvalle A, Danos O, Garcia L, van Ommen GJB, den Dunnen JT and van Deutekom JCT: Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy. Pharmacogenomics 2006; 7: 281-97. 99. Aartsma-Rus A, van Deutekom JCT, Fokkema IF, van Ommen GJC, den Dunnen JT: Entries in the Leiden DMD mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve 2006; 34: 135-44. 100. Aartsma-Rus A, Kaman WE, Weij R, den Dunnen JT, van Ommen GJB and van Deutekom JCT: Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons. Mol Ther 2006; 12: 401-7. 101. Aartsma-Rus A, de Winter CL, Janson AAM, Kamen WE, van Ommen GJB, den Dunnen JT and van Deutekom JCT: Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: Indication for steric hindrance of SR protein binding sites. Oligonucleotides 2005; 15: 285-97. 102. Bremmer-Bout M, Aartsma-Rus A, de Meijer EJ, Kaman WE, Janson A, Vossen R, van Ommen GJB, den Dunnen JT and van Deutekom JCT: Targeted exon skipping in transgenic hDMD mice: A model for direct pre-clinical screening of human-specific antisense oligonucleotides. Mol Ther 2004; 10: 232-40. 103. Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, van Ommen GJ, den Dunnen JT and van Deutekom JCT: Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet 2004; 74: 83-92. 104. Aartsma-Rus A, Kamen WE, Bremmer-Bout M, Janson A, Den Dunnen JT, van Ommen GJB and van Deutekom JCT: Comparative analysis of antisense oligonucleotides analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther 2004; 11: 1391-98. 105. Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, van Ommen GJ and van Deutekom JCT: Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genetc 2003; 12: 907-14. 106. Aartsma-Rus A, Bremmer-Bout M, Janson A, Den Dunnen J, van Ommen GJB and van Deutekom JCT: Targeted exon skipping as a potential gene correction therapy for Duchenne muscular Dystrophy. Neuromuscul Disord 2002; 12: S71-77. Other publications 1. Van Putten M and Aartsma-Rus A: Gene therapy for Duchenne muscular dystrophy. eLS (Wiley’s online reference network) 2013.
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2. Verhaart IEC and Aartsma-Rus A: Therapeutic approaches in development for Duchenne muscular dystrophy 2011; Journal for Neurology and Psychiatry of Child and Adolescent in Romania. ISSN 2068-8040. (Also translated into Romanian) 3. De Winter CL and Aartsma-Rus A: RNA-therapie voor de ziekte van Duchenne, Minder is meer. Analyse 2007; 07: 192-196. (Winner of the Anna Wichersprijs for best paper in 2007-2008) 4. Van den Bergen J, Straathof C, Aartsma-Rus A, Ginjaar HB et al: Onderzoek en zorg bij Duchenne spierdystrofie. Nederlands Tijdschrift van Geneeskunde 2009; 153:B298 Book chapters 1. Aartsma-Rus A, van Ommen GJB and Kaplan JC. (2013). Innovating therapies for muscle diseases. In Pediatric Neurology; Handbook of Clinical Neurology 3rd series (Ed. Dulac O., Lassonde M and Sarnat H) Elsevier, Chennai India. 113:1497-501. 2. Verhaart I & Aartsma-Rus A (2012) AON-mediated exon skipping for Duchenne muscular dystrophy. In Neuromuscular Disease (Ed Ashraf Zaher) Intech. 3. Spitali P & Aartsma-Rus A (2012) Duchenne muscular dystrophy: therapeutic approaches to restore dystrophin. In Muscular Disorders (Ed Madhuri Hedge) Intech. 4. Van Roon WCM & Aartsma-Rus A (2011) Overview on antisense-mediated exon skipping. In Methods of exon skipping, Methods in Molecular Biology (Ed Aartsma-Rus A) Humana Press, Springer New York Dordrecht Heidelberg London. 5. Aartsma-Rus A (2011) Antisense-mediated exon skipping for Duchenne muscular dystrophy. In Methods of exon skipping, Methods in Molecular Biology (Ed Aartsma-Rus A) Humana Press, Springer New York Dordrecht Heidelberg London. 6. Aartsma-Rus A (2011) Overview on AON design. In Methods of exon skipping, Methods in Molecular Biology (Ed Aartsma-Rus A) Humana Press, Springer New York Dordrecht Heidelberg London. 7. Aartsma-Rus A & van Ommen GJB. (2010) Antisense-mediated exon skipping for Duchenne muscular dystrophy. In Muscle Gene Therapy (Ed Duan, D) Springer New York Dordrecht Heidelberg London. 8. Aartsma-Rus A and van Deutekom JCT et al. (2009). Oligonucleotides to modulate splicing. In Methods of alternative splicing (Ed Stamm S) EURASNET Network of Excellence. In Press 9. Aartsma-Rus A and van Deutekom JCT (2008): Antisense-Mediated Reading-Frame Restoration as a Genetic Therapy for Duchenne Muscular Dystrophy. In Antisense Elements (Genetics) (Ed Hernandes, A.G.) Novapublishers, Hauppauge, NY. Edited books Methods on exon skipping in the series "Methods in Molecular Biology" by Humana Press. Patents 1. Modulation of exon recognition in pre-mrna by interfering with the secondary RNA structure (WO2004083446) Gert-Jan B van Ommen, Judith C.T. van Deutekom, Johan T den Dunnen and Annemieke AartsmaRus Publication date: September 30 2004 2. Modulation of exon recognition in pre-mRNA by interfering with the binding of SR proteins and by interfering with secondary structures (WO2006112705) Judith CT van Deutekom, Annemieke Aartsma-Rus and Gert-Jan B van Ommen Publication date: October 26, 2006 3. Means and methods for inducing exon skipping (WO2007135105) Annemieke Aartsma-Rus, Judith CT van Deutekom and Gert-Jan B van Ommen Publication date: November 29, 2007 4. Compound capable of inducing skipping of two or more exons, is that more than one exon can be skipped with a single pharmaceutical for treating many different Duchenne or Becker mutations (WO2004083446) Gert-Jan B van Ommen, Judith CT van Deutekom, Johan T den Dunnen and Annemieke AartsmaRus Publication date: January 31, 2008 5. Means and methods for counteracting muscle disorders (WO2009054725)
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Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus, Gert-Jan B van Ommen Publication date: April 30, 2009 6. Method for efficient exon (44) skipping in Duchenne muscular dystrophy and associated means (WO2009139630) Gerard Platenburg, Sjef J de Kimpe, Judith CT van Deutekom, Gert-Jan B van Ommen and Annemieke Aartsma-Rus Publication date: November 19, 2009 7. Molecule for treating an inflammatory disorder (WO 2011078672) Gert-Jan B van Ommen, Annemieke Aartsma-Rus, Judith CT van Deutekom, Sjef J de Kimpe, Sjef J Verbeek, Seda A Yilmaz-Elis Publication date: December 24, 2009 8. Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50-53 (WO2010050802) Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus and GertJan B van Ommen Publication date: May 6, 2010 9. Means and methods for efficient skipping of exon 45 of the human Duchenne muscular dystrophy pre-mRNA (WO2010050801) Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus, Gert-Jan B van Ommen Publication date: May 6, 2010 10. Means and methods for modulating notch3 protein expression and/or the coding region of notch3 (WO201085151) Saskia AJ Lesnik-Oberstein, Gert-Jan B van Ommen, Annemieke Aartsma-Rus and Hans G Dauwerse Publication date: July 29 2010 11. Antisense oligonucleotide directed removal of proteolytic cleavage sites, the HCHWA-D mutation, and trinucleotide repeat expansions (WO2012018257) Willeke van Roon-Mom, Melvin Evers, Barry Pepers, Annemieke Aartsma-Rus, Gert-Jan van Ommen Publication date August 5 2010 12. Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins (WO2012018257) Willeke van Roon-Mom, Melvin Evers, Barry Pepers, Annemieke Aartsma-Rus and Gert-Jan van Ommen Publication date August 5 2010 13. Methods and compositions for dysferlin exon-skipping (WO2011053144) Silvere M van der Maarel, Gert-Jan B van Ommen, Annemieke Aartsma-Rus, Isabella HouwelingGazzoli, Johan T den Dunnen Publication date: May 5 2011 14. Compounds and methods for altering activin receptor-like kinase signalling (WO2012138223) Peter-Barm 't Hoen, Willem Hoogaars, Dwi Kemaladewi, Annemieke Aartsma-Rus, Peter ten Dijke Publication data: April 12 2012 Meetings Functions at meetings 1. Member of the organizing committee of the 2015 TREAT-NMD International Conference. 2. Chair of the organizing committee of the 2015 Oligonucleotide Therapeutics Society meeting 3. Member of the organizing committee of the 2015 Expert Patients and Researchers Summer School (EXPRESS) in collaboration with EURORDIS 4. Organizer of the COST Training school on stakeholder communication (2015) 5. Organizer of a COST/SCOPE organized stakeholder meeting on antisense-mediated exon
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skipping therapy development for DMD, hosted by EMA (2015) 6. Member of the organizing committee of the 2015 Cold Spring Harbor Laboratory meeting on RNA and oligonucleotide therapeutics and session chair. 7. Member of the organizing committee of the 2014 Oligonucleotide Therapeutics Society meeting and session chair 8. Session Chair and discussion leader during the 2013 TREAT-NMD meeting, October 30-November 1, Newcastle, UK 9. Member of the steering committee and Session Chair of the TREAT-NMD meeting discussing the EMA draft guidelines on DMD/BMD clinical trials, June 21, London UK. 10. 16th annual meeting of the American Society of Gene and Cell Therapy, May 15-18, Salt Lake City, UT, USA: Reviewer of musculo-skeletal gene and cell therapy abstracts and chair of the Musculo-skeletal gene & cell therapy II session. 11. ENMC workshop "Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy - opportunities and challenges", Naarden, the Netherlands, Dec 2012: Coorganizer with Francesco Muntoni (UCL, London, UK) 12. 17th Worlds Muscle Society meeting, Perth, Australia, Oct 2012: Poster session facilitator and prize committee member 13. ZonMw project meeting of translational science, focuing on "EU projecten, spelers en spel", The Hague, the Netherlands, June 2012: Presented on personal experiences with translational EU projects 14. TREAT-NMD "Brining down the barriers" meeting, Geneva, Switzerland, Nov 2011: Discussion panel member 15. 16th World Muscle Society meeting, Almancil, Portugal, Oct 2011: Oral session chairperson & prize committee member 16. ZonMw workshop on Advanced Therapeutic Medicinal Projects: Hurdles and solutions in translational academic research, Heeze, the Netherlands, March 2011: Panel discussion leader 17. NIH/FDA workshop on the development of therapeutic antisense oligonucletotides for neuromuscular disorders, Washington DC, USA, Sept 2010: Steering committee member, session chairman and panel discussion leader 18. 12th International Congress on Neuromuscular disorders, Naples, Italy, July 2010: Chairman for an oral session 19. TREAT-NMD/NIH international meeting, Brussels, Belgium, Nov 2009: Organizing committee member and panel member for 2 sessions 20. Meeting with the European Medicine Agency (EMA) on The specific issues of personalized medicine in Duchenne muscular dystrophy, London, UK, Sept 2009: Member of the organizing/steering committee I have also been involved in helping to establish the scientific content in meetings organized by the Duchenne parent project onlus meeting (Italy) in 2012, 2013 and 2014, and the Duchenne parent project meeting (the Netherlands) in 2013, 2014 and 2015. Oral Presentations at scientific meetings 2015 1. SMI Conference on RNA Therapeutics, Feb 16-17 2015, London, UK: Networking towards clinical implementation of antisense-mediated exon skipping for rare diseases (invited lecture) 2. Cold Spring Harbor meeting on RNA Therapeutics, April 8-11 2015, Cold Spring Harbor, NY, USA: Beyond clinical trials for antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 3. COST /SCOPE stakeholder meeting on Translational and Regulatory challenges for DMD exon skipping development, April 29 2015, European Medicine Agency Headquarters, London, UK: DMD genetics and exon skipping 4. ExPRESS summer school, June 1 2015, Barcelona, Spain: Development of antisense-mediated exon skipping for DMD; retrospective view and lessons learnt 5. ExPRESS summer school, June 5 2015, Barcelona, Spain: COST Action BM1207 6. Meeting on 'Clinical trials and Rare Diseases, June 11, de Bilt, the Netherlands: Clinical trials for DMD; a case study (invited lecture) 7. Sarepta Therapeutics, June 22, Camebridge MA US: Biomarkers for DMD and beyond exon skipping for DMD (invited lecture)
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8. Biomarin Summit meeting, July 21-23 2015, San Francisco, CA, US: DMD phenotype, genetics and exon skipping Future lectures • PTC Masterclass on DMD, September 3-4, Amsterdam, Netherlands: Diagnostics for DMD • Biomarin Satellite Symposium at the World Muscle Society meeting, Septembee 30-October 4, Brighton UK: DMD exon skipping • Annual Oligonucleotide Therapeutics Society Conference, October 11-15, Leiden, the Netherlands: Translating exon skipping for Duchenne from Bench to Bedside (invited lecture) • EVELAM meeting, December 10-12, Lima, Peru: Dystrophinopathies (invited lecture) 2014 1. Myocon meeting, Jan 11 2014, Chennai, India: Challenges and opportunities of therapeutic approaches for Duchenne muscular dystrophy (invited lecture) 2. RD-Connect, NEUROMICS, EURENOMICS joint progress meeting, February 24-26, Heidelberg, Germany: Antisense-mediated exon skipping for DMD and other rare diseases 3. Annual meeting of the Dutch Society of Gene and Cell Therapy, March 13-14, Lunteren, the Netherlands: Antisense-mediated exon skipping: An approach with therapeutic potential for Duchenne muscular dystrophy and other diseases 4. Genetic Conference, March 27-28, Porto, Portugal: Antisense-mediated exon skipping for Duchenne muscular dystrophy – clinical trials and beyond 5. UCL seminar series, May 7, London, UK: Antisense-mediated exon skipping for Duchenne – clinical trials and beyond (invited lecture) 6. International Conference on Neuromuscular disorders, Jul 5-10 2014, Nice, France: Biomarker discovery in Duchenne muscular dystrophy 7. UCL seminar series, July 20, London, UK: Exon skipping for neuromuscular disorders (invited lecture) 8. Science Café Oct 8, Deventer, the Netherlands: Exon skippen, een manier om Duchenne patienten beter te maken 9. Education day of the European Society of Gene and Cell Therapy and the Dutch Society of Gene and Cell Therapy, Oct 23, the Hague, the Netherlands: Patient involvement in therapy development, lessons learnt from the DMD example (duo presentation with Elizabeth Vroom) (invited lecture) 10. Joint meeting of European Society of Gene and Cell Therapy and the Dutch Society of Gene and Cell Therapy, Oct 25, The Hague, the Netherlands: Beyond clinical trials for Duchenne muscular dystrophy (invited lecture) 11. Eurotides meeting, November 18-19, Berlin, Germany: Preclinical studies on Duchenne exon skipping (invited lecture) 12. John Walton institute inaugural meeting, November 24, Newcastle UK: Research and translational aspects of developing antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 13. COST Action1308 symposium on large animal models, December 15, Munich, Germany: The need for large animal models; example of antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 2013 1. Newcastle University Institute Seminars, January 29 2013, Newcastle, UK: Antisense-mediated exon skipping: a promising therapeutic approach for Duchenne muscular dystrophy and other rare diseases (invited lecture) 2. Workshop on FSHD trial readiness, April 8-9 2013, Leiden, the Netherlands: TREAT-NMD and patient registries (invited lecture) 3. International conference of the IRDiRC, April 15-17 2013, Dublin, Ireland: Antisense-mediated exon skipping – Applying lessons learnt from Duchenne muscular dystrophy to other rare diseases (invited lecture) 4. Children’s National Medical Center, April 24 2013, Washington DC: the development of antisensemediated exon skipping for DMD (invited lecture) 5. ASGCT satellite meeting of the Gene and Cell Therapy Consortium, May 17 2013, Salt Lake City, UT, USA: the TREAT-NMD paradigm (invited lecture) 6. Tanjin University (China), August 22 2013: Antisense-mediated exon skipping for DMD (invited
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lecture) 7. Gene therapy workshop, Sept 13-15 2013, Leiden, the Netherlands: Opportunities of antisensemediated exon skipping (invited lecture) 8. Rare Disease symposium of the Italian Pharmacological Society, Oct 23-26 2013, Turin, Italy: Antisense-mediated exon skipping – a therapeutic approach with potential for DMD and other rare diseases (invited lecture) 9. TREAT-NMD symposium, Oct 29-Nov 1 2013, Newcastle UK: Joint research and regulatory interaction (invited lecture) 10. Annual meeting of the Finish Society of Pediatric Neurology in memory of Märtha Donner, Nov 7, Helsinki, Finland: Preclinical development of antisense-mediated exon skipping for DMD and other neurologic diseases (invited lecture) 11. Cardiovascular genetics symposium, Nov 20-22 2013, Cardiff, UK: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 12. Duchenne Policy Forum, Dec 12 2013, Washington DC, USA: EMA draft guidelines for testing medicinal products in DMD and BMD patients 13. Leiter des Schwerpunkts Klinische Neuroonkologie lecture, Dec 17, Bonn, Germany: Antisensemediated exon skipping for Duchenne muscular dystrophy and other neurological disorders (invited lecture) 2012 1. The Israeli Neuromuscular Conference, Feb 27 2012, Holon, Israel: Therapeutic approaches in development for Duchenne muscular dystrophy (invited lecture) 2. Rare Disease meeting Orphanet-Israel, Feb 29 2012, Schneider Hospital, Israel: Exon skipping as a potential therapy for Duchenne muscular dystrophy (invited lecture) 3. Muscular Dystrophy Association Clinical Conference, March 4-7 2012, Las Vegas, USA: Antisensemediated exon skipping for Duchenne muscular dystrophy (invited lecture) 4. 6th European conference on Rare Diseases & Orphan Products, May 23-25 2012, Brussels, Belgium: AON applications in Rare Diseases (invited lecture) 5. 17th World Muscle Society Meeting, Oct 9-14 2012, Perth, Australia: DMD transcript imbalance regulates dystrophin levels. 6. Eurotides meeting, November 26-28 2012, Berlin, Germany: Preclinical studies on exon skipping for Duchenne muscular dystrophy (invited lecture) 7. Combined NEUROMICS, EuRenOmics, RD-Connect kick-off meeting, January 25-27 2012, Barcelona, Spain: Exon skipping therapy development: the future, past and present. 2011 1. Nijmegen Academic Medical Center Masterclass on genetic therapy, Jan 17-21 2011, Nijmegen, the Netherlands: Antisense-oligonucleotide treatment for Duchenne muscular dystrophy (invited lecture). 2. Standards of care in managing people with DMD, March 18-20 2011, Bucharest, Romania: Current and future therapies (invited lecture) 3. Congress of the German Society of Muscle disease, March 30 – April 2 2011, Ulm, Germany: The principles and perspectives for antisense-mediated exon skipping for DMD (invited lecture) 4. 1st Chinese conference on translational research in Duchenne muscular dystrophy, April 7-9 2011, Guanzhou, China: Therapeutic concepts based on RNA and DNA modifications in DMD (invited lecture) 5. Lecture series of the Groningen University Medical Center, April 25 2011, Groningen, the Netherlands: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture). 6. 14th Annual meeting of the American Society of Gene and Cell Therapy, May 18-21 2011, Seattle WA, USA: Learning from exon skipping trials in Duchenne muscular dystrophy (invited lecture) 7. Amsterdam Medical Therapeutics, June 17 2011, Amsterdam, the Netherlands: How much dystrophin is needed for improvement of Duchenne muscular dystrophy (invited lecture) 8. Maastricht scientific symposium, June 28 2011, Maastricht, the Netherlands: Development of Antisense-mediated exon skipping Therapy for Duchenne Muscular Dystrophy (Keynote lecture) 9. Brickless symposium of Scandinavian Neurologists, Sept 7 2011, Odense, Denmark: Future treatments for patients with NMDs (invited lecture) 10. International EU-AxRegen Workshop Amsterdam, Sept 27-29 2011, Amsterdam, the Netherlands:
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Developing a therapy for Duchenne Muscular Dystrophy (DMD) based on exon-skipping (Invited lecture) 11. 16th annual meeting of the world muscle society, Oct 18-22 2011, Almancil, Portugal: Development of systemic antisense treatment in dystrophic mouse models for Duchenne Muscular Dystrophy 12. Cold Spring Harbor Laboratory course on RNA Therapeutics, Dec 4-7 2011, Cold Spring Harbor, NY, USA: Development of systemic antisense treatment in dystrophic mouse models for Duchenne muscular dystrophy (invited lecture) 2010 1. Nederlandse Vereniging van Gen Therapie, March 11 2010, Heuze, the Netherlands: Exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 2. Nederlandse Vereniging van Gen Therapie, March 12 2010, Heuze, the Netherlands: Antisensemediated exon skipping as a therapy for Duchenne muscular dystrophy 3. Cold spring harbor meeting RNA and oligonucleotides therapeutics, April 7-10 2010, Cold Spring Harbor, NY, USA: Long term systemic antisense-mediated exon skipping in dystrophic mouse models. 4. Belgium meeting of Child Neurology, April 24 2010, Gent, Belgium: Use of antisense oligonucleotides. 5. International Translational Research Conference for NMDs, June 17-19 2010, Sao Paulo, Brazil: Innovative therapeutic approaches for DMD (invited lecture) 6. EURASNET mRNA and mis-splicing: disease and therapeutics, July 13-15 2010, Cambridge, UK: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 7. 12th international congress on neuromuscular diseases, July 17-22 2010, Naples, Italy: Learning from early trials of antisense oligonucleotide therapy for DMD (invited lecture) 8. NIH/FDA Conference on Antisense Oligonucleotide Therapies in Neuromuscular Disease, September 27 2010, Washington DC, USA: TBA 9. Seminar series of the Nationwide Children’s Hospital, Columbus (OH), USA, September 30 2010: Antisense oligonucleotides treatment for neuromuscular and neurological disorders 10. European Society of Gene and Cell Therapy, Educational session, October 22 2010, Milan, Italy: Antisense-mediated exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 11. Portuguese Human Genetics Conference, November 18-20 2010, Coimbra, Portugal: Development of antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 2009 1. Keystone meeting on Therapeutic Modulation of RNA Using Oligonucleotides, Feb 8-13 2009, Lake Louise (AB) Canada: Clinical trials for antsisense-mediated exon skipping for Duchenne Muscular Dystrophy (invited lecture) 2. Nederlandse Vereniging van Humane Genetica meeting, 23-24 April 2009, Veldhoven the Netherlands: First clinical trial on therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 3. Meeting Molecular Therapeutics of the Interdisciplinary Centre for Clinical Research (IZKF) of the University of Erlängen (Germany), 14-16 May 2009, Nürnberg Germany: Antisense-mediated exon skipping: preclinical and clinical development of a therapy for Duchenne muscular dystrophy (invited lecture) 4. Lecture at AVI-Biopharma, Corvallis, USA, May 26 2009: Development of antisense-mediated exon skipping for Duchenne muscular dystrophy – from design to clinical trials (invited lecture) 5. 2009 annual meeting of the American Society of Gene Therapy, May 29 2009: Systemic AntisenseMediated Exon Skipping in Mouse Models for Duchenne Muscular Dystrophy 6. Francophone Society of Cell and Gene Therapy Meeting, 21-23 June 2009, Paris France: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 7. Oxford University, July 9 2009, Oxford UK: Development of antisense-mediated exon skipping for Duchenne muscular dystrophy; from design to clinical trials (invited lecture) 8. Workshop on alternative splicing and disease, Montpellier, France, July 20 2009: Antisensemediated exon skipping therapy for Duchenne muscular dystrophy – design, preclinical studies and clinical trials (invited lecture) 9. Cold Spring Harbor Laboratories, August 17 2009, Cold Spring Harbor, NY, USA: Development of antisense-mediated exon skipping therapy for Duchenne muscular dystrophy – from proof of concept to preclinical and clinical trials (invited lecture)
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10. EMEA headquarters, September 25 2009. TREAT-NMD workshop “The development of antisense oligonucleotide therapies for Duchenne Muscular Dystrophy”: The specific issues of personalized medicine in Duchenne muscular dystrophy 11. Joint meeting of the oligonucleotide therapeutic society and antisense society Japan, workshop “oligos targeting DMD and diseases involving splicing” 3-6 Nov 2009, Fukuota, Japan; speaker (long term systemic studies with antisense oligonucleotides in Duchenne muscular dystrophy models) and panel member 12. International symposium on DMD exon skipping, Nov 10 2009, Kobe, Japan: Development of exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 13. Eurotides meeting, December 3 2009, Amsterdam, the Netherlands: Preclinical development of systemic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 2005-2008 1. Séminaire Généthon, February 24 2005, Evry, France: Development of an antisense-mediated exon-skipping therapy for Duchenne Muscular Dystrophy (invited lecture) 2. ESF and ICGDB joint workshop on The pathology of pre-mRNA splicing: diagnostic and mechanistic aspects, April 7-9 2005, Trieste, Italy: Blocking SR binding sites by antisense oligonucleotides to indice exon skipping: therapeutic applications for Duchenne Muscular Dystrophy 3. Symposium on Alternate Transcript Diversity II - Biology, and Therapeutics, March 21-23, 2006, Heidelberg, Germany: Therapeutic antisense-mediated exon skipping for Duchenne Muscular Dystrophy (invited lecture) 4. 9th Annual meeting of the American Society of Gene Therapy, May 31 - June 4, 2006, Baltimore, USA: First-in-Man Study on Antisense-Induced Exon Skipping for Duchenne Muscular Dystrophy (invited lecture) 5. 28e congres Kindergeneeskunde (Pediatrics), November 1-3, 2006, Veldhoven, The Netherlands: Antisense-gemedieerde exon skip therapie voor Duchenne Spierdystrofie (invited lecture) 6. Eurostemcell clinical workshop: Towards clinical trials in Duchenne Muscular Dystrophy, April 3-4, 2007, Bellagio, Italy: Towards clinical application of antisense-mediated exon skipping for DMD (invited lecture) 7. 7th National Congress, Italian Association for Myology, May 25-26 2007, Ferrara, Italy: The development of antisense-mediated exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 8. 12th annual meeting of the RNA Society, May 28 - June 3 2007, Madison, USA: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy 9. 2007 Annual meeting of the American Society of Human Genetics 23-27 October 2007, San Diego USA: Antisense-mediated exon 51 skipping restores local dystrophin expression in muscle of Duchenne muscular dystrophy patients 10. 15th Meeting of the European Society of Gene and Cell Therapy 27-30 October 2007, Rotterdam The Netherlands: In vivo optimization of therapuetic antisense-mediated exon skipping for Duchenne muscular dystrophy 11. European conference of human genetics meeting 2008, 31 May - 3 June, Barcelona Spain: Systemic antisense-mediated exon skipping studies in mouse models for Duchenne muscular dystrophy 12. First international meeting of the EURASNET consortium, on alternative splicing 21-23 May 2008, Krakaw Poland: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 13. 13th annual meeting of the RNA Society, July 28 - August 3 2008, Berlin, Germany: First clinical trial on therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy 14. Neurotherapy Summerschool of brain research (Dutch Institute of Neuroscience), August 25-28, Amsterdam, the Netherlands: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture; replacing Mimoun Azzouz) 15. Fourth annual meeting of the oligonucleotide therapeutic society, 15-18 October 2008, Boston (MA) USA: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) Projects
Grants as (co)PI 1. Duchenne Parent Project grant 2014-2015
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Improving 2’-O-methyl phosphorothioate induced exon skipping for DMD by modified chemistry and/or combination with adjunct compounds €240,000 Role, applicant, PI 2. Prinses Beatrix Spierfonds Identification of cyclic peptides to improve muscle and heart uptake of AONs 2013-2015 €250,000 Role: applicant, PI 3. Duchenne Parent Project – small grant DMD liaison post at TREAT-NMD office €25,000 funding to cover 6 months salary 4. COST Action “Networking towards clinical application of antisense-mediated exon skipping for rare diseases” 2013-2016 Networking grant for up to €400,000 Role: applicant, Action Chair 5. Duchenne Parent Project The effect of different types of exercise in mice with low levels of dystrophin Jan 2013-Dec 2014 €250,000 Role: applicant, PI 3. ZonMw Antisense therapy for several major rare diseases Jan 2013 -Dec 2016 €3,000,000 Role: co-applicant, PI 4. NEUROMICS (Integrated European Project on Omics Research of Rare Neuromuscular and Neurodegenerative Diseases) 2012-2016 €750,000 (from 12 million euro for grant) Role: work package leader 5. Dutch Duchenne Parent Project Long term treatment effects of antisense-mediated exon skipping in Duchenne muscular dystrophy 2011-2013 (2 years) €222,000 Role: PI 6. BIO-NMD FP7 EU Jan 2010 – Dec 2012 (3 years) €780,000 Role: Partner 7. VIDI Grant Dissecting the splicing process of the 2.4 Mb DMD gene by transcript deep sequencing to optimize antisense-mediated exon skipping for Duchenne muscular dystrophy 2010-2014 (5 years) €800,000 Role: PI 8. PBF Grant (Prinses Beatrix Foundation)
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Dual treatment approaches for Duchenne muscular dystrophy 2010-2014 (4 years) €250,000 Role: PI 9. Aspasia Grant (NWO, Dutch Science Organization) €100,000 Special grant to promote the number of females in associate and full professor positions. Role: PI 10. ZonMw Pilot Program Translational Research (Co-application with Gert-Jan van Ommen) Development of an innovative monitoring technology to guide the in vivo systemic treatment efficacy of antisense-exon skipping gene-correction therapy 2009-2010 (2 years) €462,032 Role: PI 11. Horizon Grant Dissecting the splicing process of the 2.4 Mb DMD gene by deep sequencing 2009-2010 (1.5 years) €100,000 Role: PI 12. EU FP6 (Network of excellence) TREAT-NMD (Translational research to accelerate treatments for neuromuscular disorders) Jan 2007 – Dec 2011 €775,000 (from 10 million euro for Network) Role: workpackage leader 13. Dutch Duchenne Parent Project Optimization of (long-term) systemic delivery and uptake of antisense oligonucleotides and conjugates in different DMD mouse models Sept 2008 – Aug 2010 €312,193 Role: PI Co-applications 14. Dutch Duchenne Parent Project Elucidating the role and therapeutic potential of myostatin/TGF-β receptors Cripto and ALK1 in skeletal muscle regeneration and DMD pathology Jan 2013 – Dec 2014 €160,000 Applicant Dr. Willem Hoogaars, Role: supervisor 15. E-Rare Splice-EB 2013-2016 €850,000 Applicant Marjon Pasmooij, Role: co-applicant, advisor 16. Dutch Duchenne Parent Project An inducible dystrophin mouse model 2012-2014 €190,000 Role: Supervisor; PI: Dr. M Veltrop (LUMC) 17. Dutch Duchenne Parent Project
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Targeting of TGFβ cascades in mdx mice to improve muscle quality 2011-2013 €175,000 Role: Supervisor; PI: Dr. W Hoogaars (LUMC) 18. MDA AONs to modulate the transcriptional landscape of D4Z4 $200,000 2010-2012 (2 years) Role: collaborator, PI: Prof. S. van der Maarel (LUMC, the Netherlands) 19. Hersenstichting Cysteine quantity correction in CADASIL €150,000 2010-2012 (2 years) Role: collaborator, PI: Dr. S. Lesnik Oberstein (LUMC, the Netherlands) 20. BIO-NMD FP7 EU €780,000 to LUMC 2010-2012 (3 years) Role: partner, PI Alessandra Ferlini (University of Ferrara, Italy) 21. Prinses Beatrix Foundation Integrated molecular and clinical research towards a therapy for DMD €450,000 2008-2012 Role: coordinator, PI Gert-Jan van Ommen (LUMC, the Netherlands) 22. IOP Genomics Towards broad clinical and technological application of gene expression engineering by exon skipping €1,600,000 2007-2012 Role: collaborator, PI: Gert-Jan van Ommen (LUMC, the Netherlands) Memberships
2Current Since 2015 Member of the IRDiRC Task force on patient centered outcome measures Since 2014 Member of the junior Dutch Royal Academy of Sciences Since 2014 Leader of the “Science and Society” section of the junior Dutch Royal Academy of Sciences Since 2014 Member of the editorial board of Nucleic Acid Therapeutics Since 2014 Member of the editorial board of Cardiovascular Genetics and Genomics Since 2014 Extended Committee Member of the TREAT-NMD Advisory Committee on Therapeutics (TACT) Since 2013 Associate editor of the Journal of Neuromuscular Diseases 2013-2017 Chair of the COST Action BM1207 (Networking towards clinical implementation of antisense-mediated exon skipping for rare diseases) 2013-2015 Chair of the TREAT-NMD alliance 2013-2015 Chair of the planning committee for the 2015 annual meeting of the oligonucleotide therapeutics society (the Netherlands) 2013-2015 Member of the Scientific Counsel “Molecular and Pathophysiological basis of Muscular Dystrophies” of the Association Française contre les Myopathies Since 2010 Member of the editorial board of the World Journal of Medical Genetics Past
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2014 Member of the working groups involved in drafting the biomarker part and the diagnosis part of a policy guidance for the U.S. Food and Drug Administration on clinical trials in DMD patients 2013-2014 Member of the planning committee for the 2014 annual meeting of the oligonucleotide therapeutic society (San Diego, USA) 2013-2014 Member of the Advanced Therapies Working Group of the International Rare Disease Research Consortium (IRDiRC) 2013-2014 Member and Secretary of the Board of Directors of the Oligonucleotides Therapeutics Society 2013 Member of the steering committee for the organization of a TREAT-NMD/COST/EMA meeting on guidelines for clinical trials in D/BMD patients 2013 Member of the Steering Group for workshops about the EMA guidelines for “Therapies for DMD and BMD patients” 2012–2013 Vice chair of the executive board of the TREAT-NMD alliance 2011 Member of the Task Force of the TREAT-NMD alliance 2011–2012 Board member of the Leiden Center for Translational Neurosciences 2010 Member of the steering committee for the FDA workshop on the development of therapeutic antisense oligonucleotides for neuromuscular diseases 2009 Organizing committee for the TREAT-NMD/NIH international meeting member 2008 – 2009 Member of the steering committee for the EMEA Workshop on Trial Protocol Design for Regulatory Approval of AON Therapies in DMD 2007 – present Member of the Project Ethics Counsel (TREAT-NMD network of excellence) Memberships of international societies Member of the Oligonucleotide Therapeutic Society since 2009 Member of the World Muscle Society since 2007 Member of the American Society of Gene and Cell Therapy since 2010 Other publications 1. Van Putten M and Aartsma-Rus A: Gene therapy for Duchenne muscular dystrophy. eLS (Wiley’s online reference network) 2013. 2. Verhaart IEC and Aartsma-Rus A: Therapeutic approaches in development for Duchenne muscular dystrophy 2011; Journal for Neurology and Psychiatry of Child and Adolescent in Romania. ISSN 2068-8040. (Also translated into Romanian) 3. De Winter CL and Aartsma-Rus A: RNA-therapie voor de ziekte van Duchenne, Minder is meer. Analyse 2007; 07: 192-196. (Winner of the Anna Wichersprijs for best paper in 2007-2008) 4. Van den Bergen J, Straathof C, Aartsma-Rus A, Ginjaar HB et al: Onderzoek en zorg bij Duchenne spierdystrofie. Nederlands Tijdschrift van Geneeskunde 2009; 153:B298 Book chapters 1. Aartsma-Rus A, van Ommen GJB and Kaplan JC. (2013). Innovating therapies for muscle diseases. In Pediatric Neurology; Handbook of Clinical Neurology 3rd series (Ed. Dulac O., Lassonde M and Sarnat H) Elsevier, Chennai India. 113:1497-501. 2. Verhaart I & Aartsma-Rus A (2012) AON-mediated exon skipping for Duchenne muscular dystrophy. In Neuromuscular Disease (Ed Ashraf Zaher) Intech. 3. Spitali P & Aartsma-Rus A (2012) Duchenne muscular dystrophy: therapeutic approaches to restore dystrophin. In Muscular Disorders (Ed Madhuri Hedge) Intech. 4. Van Roon WCM & Aartsma-Rus A (2011) Overview on antisense-mediated exon skipping. In Methods of exon skipping, Methods in Molecular Biology (Ed Aartsma-Rus A) Humana Press, Springer New York Dordrecht Heidelberg London. 5. Aartsma-Rus A (2011) Antisense-mediated exon skipping for Duchenne muscular dystrophy. In Methods of exon skipping, Methods in Molecular Biology (Ed Aartsma-Rus A) Humana Press, Springer New York Dordrecht Heidelberg London. 6. Aartsma-Rus A (2011) Overview on AON design. In Methods of exon skipping, Methods in Molecular Biology (Ed Aartsma-Rus A) Humana Press, Springer New York Dordrecht Heidelberg London. 7. Aartsma-Rus A & van Ommen GJB. (2010) Antisense-mediated exon skipping for Duchenne muscular dystrophy. In Muscle Gene Therapy (Ed Duan, D) Springer New York Dordrecht Heidelberg
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London. 8. Aartsma-Rus A and van Deutekom JCT et al. (2009). Oligonucleotides to modulate splicing. In Methods of alternative splicing (Ed Stamm S) EURASNET Network of Excellence. In Press 9. Aartsma-Rus A and van Deutekom JCT (2008): Antisense-Mediated Reading-Frame Restoration as a Genetic Therapy for Duchenne Muscular Dystrophy. In Antisense Elements (Genetics) (Ed Hernandes, A.G.) Novapublishers, Hauppauge, NY. Edited books Methods on exon skipping in the series "Methods in Molecular Biology" by Humana Press. Patents 1. Modulation of exon recognition in pre-mrna by interfering with the secondary RNA structure (WO2004083446) Gert-Jan B van Ommen, Judith C.T. van Deutekom, Johan T den Dunnen and Annemieke AartsmaRus Publication date: September 30 2004 2. Modulation of exon recognition in pre-mRNA by interfering with the binding of SR proteins and by interfering with secondary structures (WO2006112705) Judith CT van Deutekom, Annemieke Aartsma-Rus and Gert-Jan B van Ommen Publication date: October 26, 2006 3. Means and methods for inducing exon skipping (WO2007135105) Annemieke Aartsma-Rus, Judith CT van Deutekom and Gert-Jan B van Ommen Publication date: November 29, 2007 4. Compound capable of inducing skipping of two or more exons, is that more than one exon can be skipped with a single pharmaceutical for treating many different Duchenne or Becker mutations (WO2004083446) Gert-Jan B van Ommen, Judith CT van Deutekom, Johan T den Dunnen and Annemieke AartsmaRus Publication date: January 31, 2008 5. Means and methods for counteracting muscle disorders (WO2009054725) Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus, Gert-Jan B van Ommen Publication date: April 30, 2009 6. Method for efficient exon (44) skipping in Duchenne muscular dystrophy and associated means (WO2009139630) Gerard Platenburg, Sjef J de Kimpe, Judith CT van Deutekom, Gert-Jan B van Ommen and Annemieke Aartsma-Rus Publication date: November 19, 2009 7. Molecule for treating an inflammatory disorder (WO 2011078672) Gert-Jan B van Ommen, Annemieke Aartsma-Rus, Judith CT van Deutekom, Sjef J de Kimpe, Sjef J Verbeek, Seda A Yilmaz-Elis Publication date: December 24, 2009 8. Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50-53 (WO2010050802) Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus and GertJan B van Ommen Publication date: May 6, 2010 9. Means and methods for efficient skipping of exon 45 of the human Duchenne muscular dystrophy pre-mRNA (WO2010050801) Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus, Gert-Jan B van Ommen Publication date: May 6, 2010 10. Means and methods for modulating notch3 protein expression and/or the coding region of notch3 (WO201085151)
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Saskia AJ Lesnik-Oberstein, Gert-Jan B van Ommen, Annemieke Aartsma-Rus and Hans G Dauwerse Publication date: July 29 2010 11. Antisense oligonucleotide directed removal of proteolytic cleavage sites, the HCHWA-D mutation, and trinucleotide repeat expansions (WO2012018257) Willeke van Roon-Mom, Melvin Evers, Barry Pepers, Annemieke Aartsma-Rus, Gert-Jan van Ommen Publication date August 5 2010 12. Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins (WO2012018257) Willeke van Roon-Mom, Melvin Evers, Barry Pepers, Annemieke Aartsma-Rus and Gert-Jan van Ommen Publication date August 5 2010 13. Methods and compositions for dysferlin exon-skipping (WO2011053144) Silvere M van der Maarel, Gert-Jan B van Ommen, Annemieke Aartsma-Rus, Isabella HouwelingGazzoli, Johan T den Dunnen Publication date: May 5 2011 14. Compounds and methods for altering activin receptor-like kinase signalling (WO2012138223) Peter-Barm 't Hoen, Willem Hoogaars, Dwi Kemaladewi, Annemieke Aartsma-Rus, Peter ten Dijke Publication data: April 12 2012 Meetings Functions at meetings 1. Member of the organizing committee of the 2015 TREAT-NMD International Conference. 2. Chair of the organizing committee of the 2015 Oligonucleotide Therapeutics Society meeting 3. Member of the organizing committee of the 2015 Expert Patients and Researchers Summer School (EXPRESS) in collaboration with EURORDIS 4. Organizer of the COST Training school on stakeholder communication (2015) 5. Organizer of a COST/SCOPE organized stakeholder meeting on antisense-mediated exon skipping therapy development for DMD, hosted by EMA (2015) 6. Member of the organizing committee of the 2015 Cold Spring Harbor Laboratory meeting on RNA and oligonucleotide therapeutics and session chair. 7. Member of the organizing committee of the 2014 Oligonucleotide Therapeutics Society meeting and session chair 8. Session Chair and discussion leader during the 2013 TREAT-NMD meeting, October 30-November 1, Newcastle, UK 9. Member of the steering committee and Session Chair of the TREAT-NMD meeting discussing the EMA draft guidelines on DMD/BMD clinical trials, June 21, London UK. 10. 16th annual meeting of the American Society of Gene and Cell Therapy, May 15-18, Salt Lake City, UT, USA: Reviewer of musculo-skeletal gene and cell therapy abstracts and chair of the Musculo-skeletal gene & cell therapy II session. 11. ENMC workshop "Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy - opportunities and challenges", Naarden, the Netherlands, Dec 2012: Coorganizer with Francesco Muntoni (UCL, London, UK) 12. 17th Worlds Muscle Society meeting, Perth, Australia, Oct 2012: Poster session facilitator and prize committee member 13. ZonMw project meeting of translational science, focuing on "EU projecten, spelers en spel", The Hague, the Netherlands, June 2012: Presented on personal experiences with translational EU projects 14. TREAT-NMD "Brining down the barriers" meeting, Geneva, Switzerland, Nov 2011: Discussion panel member 15. 16th World Muscle Society meeting, Almancil, Portugal, Oct 2011: Oral session chairperson & prize committee member 16. ZonMw workshop on Advanced Therapeutic Medicinal Projects: Hurdles and solutions in translational academic research, Heeze, the Netherlands, March 2011: Panel discussion leader
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17. NIH/FDA workshop on the development of therapeutic antisense oligonucletotides for neuromuscular disorders, Washington DC, USA, Sept 2010: Steering committee member, session chairman and panel discussion leader 18. 12th International Congress on Neuromuscular disorders, Naples, Italy, July 2010: Chairman for an oral session 19. TREAT-NMD/NIH international meeting, Brussels, Belgium, Nov 2009: Organizing committee member and panel member for 2 sessions 20. Meeting with the European Medicine Agency (EMA) on The specific issues of personalized medicine in Duchenne muscular dystrophy, London, UK, Sept 2009: Member of the organizing/steering committee I have also been involved in helping to establish the scientific content in meetings organized by the Duchenne parent project onlus meeting (Italy) in 2012, 2013 and 2014, and the Duchenne parent project meeting (the Netherlands) in 2013, 2014 and 2015. Oral Presentations at scientific meetings 2015 1. SMI Conference on RNA Therapeutics, Feb 16-17 2015, London, UK: Networking towards clinical implementation of antisense-mediated exon skipping for rare diseases (invited lecture) 2. Cold Spring Harbor meeting on RNA Therapeutics, April 8-11 2015, Cold Spring Harbor, NY, USA: Beyond clinical trials for antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 3. COST /SCOPE stakeholder meeting on Translational and Regulatory challenges for DMD exon skipping development, April 29 2015, European Medicine Agency Headquarters, London, UK: DMD genetics and exon skipping 4. ExPRESS summer school, June 1 2015, Barcelona, Spain: Development of antisense-mediated exon skipping for DMD; retrospective view and lessons learnt 5. ExPRESS summer school, June 5 2015, Barcelona, Spain: COST Action BM1207 6. Meeting on 'Clinical trials and Rare Diseases, June 11, de Bilt, the Netherlands: Clinical trials for DMD; a case study (invited lecture) 7. Sarepta Therapeutics, June 22, Camebridge MA US: Biomarkers for DMD and beyond exon skipping for DMD (invited lecture)Biomarin Summit meeting, July 21-23 2015, San Francisco, CA, US: DMD phenotype, genetics and exon skippingPTC Masterclass on DMD, September 3-4, Amsterdam, Netherlands: Diagnostics for DMD 8. Biomarin Satellite Symposium at the World Muscle Society meeting, Septembee 30-October 4, Brighton UK: DMD exon skipping 9. Annual Oligonucleotide Therapeutics Society Conference, October 11-15, Leiden, the Netherlands: Translating exon skipping for Duchenne from Bench to Bedside (invited lecture) Future presentations • EVELAM meeting, December 10-12, Lima, Peru: Dystrophinopathies (invited lecture) 2014 1. Myocon meeting, Jan 11 2014, Chennai, India: Challenges and opportunities of therapeutic approaches for Duchenne muscular dystrophy (invited lecture) 2. RD-Connect, NEUROMICS, EURENOMICS joint progress meeting, February 24-26, Heidelberg, Germany: Antisense-mediated exon skipping for DMD and other rare diseases 3. Annual meeting of the Dutch Society of Gene and Cell Therapy, March 13-14, Lunteren, the Netherlands: Antisense-mediated exon skipping: An approach with therapeutic potential for Duchenne muscular dystrophy and other diseases 4. Genetic Conference, March 27-28, Porto, Portugal: Antisense-mediated exon skipping for Duchenne muscular dystrophy – clinical trials and beyond 5. UCL seminar series, May 7, London, UK: Antisense-mediated exon skipping for Duchenne – clinical trials and beyond (invited lecture) 6. International Conference on Neuromuscular disorders, Jul 5-10 2014, Nice, France: Biomarker discovery in Duchenne muscular dystrophy 7. UCL seminar series, July 20, London, UK: Exon skipping for neuromuscular disorders (invited lecture) 8. Science Café Oct 8, Deventer, the Netherlands: Exon skippen, een manier om Duchenne patienten
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beter te maken 9. Education day of the European Society of Gene and Cell Therapy and the Dutch Society of Gene and Cell Therapy, Oct 23, the Hague, the Netherlands: Patient involvement in therapy development, lessons learnt from the DMD example (duo presentation with Elizabeth Vroom) (invited lecture) 10. Joint meeting of European Society of Gene and Cell Therapy and the Dutch Society of Gene and Cell Therapy, Oct 25, The Hague, the Netherlands: Beyond clinical trials for Duchenne muscular dystrophy (invited lecture) 11. Eurotides meeting, November 18-19, Berlin, Germany: Preclinical studies on Duchenne exon skipping (invited lecture) 12. John Walton institute inaugural meeting, November 24, Newcastle UK: Research and translational aspects of developing antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 13. COST Action1308 symposium on large animal models, December 15, Munich, Germany: The need for large animal models; example of antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 2013 1. Newcastle University Institute Seminars, January 29 2013, Newcastle, UK: Antisense-mediated exon skipping: a promising therapeutic approach for Duchenne muscular dystrophy and other rare diseases (invited lecture) 2. Workshop on FSHD trial readiness, April 8-9 2013, Leiden, the Netherlands: TREAT-NMD and patient registries (invited lecture) 3. International conference of the IRDiRC, April 15-17 2013, Dublin, Ireland: Antisense-mediated exon skipping – Applying lessons learnt from Duchenne muscular dystrophy to other rare diseases (invited lecture) 4. Children’s National Medical Center, April 24 2013, Washington DC: the development of antisensemediated exon skipping for DMD (invited lecture) 5. ASGCT satellite meeting of the Gene and Cell Therapy Consortium, May 17 2013, Salt Lake City, UT, USA: the TREAT-NMD paradigm (invited lecture) 6. Tanjin University (China), August 22 2013: Antisense-mediated exon skipping for DMD (invited lecture) 7. Gene therapy workshop, Sept 13-15 2013, Leiden, the Netherlands: Opportunities of antisensemediated exon skipping (invited lecture) 8. Rare Disease symposium of the Italian Pharmacological Society, Oct 23-26 2013, Turin, Italy: Antisense-mediated exon skipping – a therapeutic approach with potential for DMD and other rare diseases (invited lecture) 9. TREAT-NMD symposium, Oct 29-Nov 1 2013, Newcastle UK: Joint research and regulatory interaction (invited lecture) 10. Annual meeting of the Finish Society of Pediatric Neurology in memory of Märtha Donner, Nov 7, Helsinki, Finland: Preclinical development of antisense-mediated exon skipping for DMD and other neurologic diseases (invited lecture) 11. Cardiovascular genetics symposium, Nov 20-22 2013, Cardiff, UK: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 12. Duchenne Policy Forum, Dec 12 2013, Washington DC, USA: EMA draft guidelines for testing medicinal products in DMD and BMD patients 13. Leiter des Schwerpunkts Klinische Neuroonkologie lecture, Dec 17, Bonn, Germany: Antisensemediated exon skipping for Duchenne muscular dystrophy and other neurological disorders (invited lecture) 2012 1. The Israeli Neuromuscular Conference, Feb 27 2012, Holon, Israel: Therapeutic approaches in development for Duchenne muscular dystrophy (invited lecture) 2. Rare Disease meeting Orphanet-Israel, Feb 29 2012, Schneider Hospital, Israel: Exon skipping as a potential therapy for Duchenne muscular dystrophy (invited lecture) 3. Muscular Dystrophy Association Clinical Conference, March 4-7 2012, Las Vegas, USA: Antisensemediated exon skipping for Duchenne muscular dystrophy (invited lecture) 4. 6th European conference on Rare Diseases & Orphan Products, May 23-25 2012, Brussels, Belgium: AON applications in Rare Diseases (invited lecture)
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5. 17th World Muscle Society Meeting, Oct 9-14 2012, Perth, Australia: DMD transcript imbalance regulates dystrophin levels. 6. Eurotides meeting, November 26-28 2012, Berlin, Germany: Preclinical studies on exon skipping for Duchenne muscular dystrophy (invited lecture) 7. Combined NEUROMICS, EuRenOmics, RD-Connect kick-off meeting, January 25-27 2012, Barcelona, Spain: Exon skipping therapy development: the future, past and present. 2011 1. Nijmegen Academic Medical Center Masterclass on genetic therapy, Jan 17-21 2011, Nijmegen, the Netherlands: Antisense-oligonucleotide treatment for Duchenne muscular dystrophy (invited lecture). 2. Standards of care in managing people with DMD, March 18-20 2011, Bucharest, Romania: Current and future therapies (invited lecture) 3. Congress of the German Society of Muscle disease, March 30 – April 2 2011, Ulm, Germany: The principles and perspectives for antisense-mediated exon skipping for DMD (invited lecture) 4. 1st Chinese conference on translational research in Duchenne muscular dystrophy, April 7-9 2011, Guanzhou, China: Therapeutic concepts based on RNA and DNA modifications in DMD (invited lecture) 5. Lecture series of the Groningen University Medical Center, April 25 2011, Groningen, the Netherlands: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture). 6. 14th Annual meeting of the American Society of Gene and Cell Therapy, May 18-21 2011, Seattle WA, USA: Learning from exon skipping trials in Duchenne muscular dystrophy (invited lecture) 7. Amsterdam Medical Therapeutics, June 17 2011, Amsterdam, the Netherlands: How much dystrophin is needed for improvement of Duchenne muscular dystrophy (invited lecture) 8. Maastricht scientific symposium, June 28 2011, Maastricht, the Netherlands: Development of Antisense-mediated exon skipping Therapy for Duchenne Muscular Dystrophy (Keynote lecture) 9. Brickless symposium of Scandinavian Neurologists, Sept 7 2011, Odense, Denmark: Future treatments for patients with NMDs (invited lecture) 10. International EU-AxRegen Workshop Amsterdam, Sept 27-29 2011, Amsterdam, the Netherlands: Developing a therapy for Duchenne Muscular Dystrophy (DMD) based on exon-skipping (Invited lecture) 11. 16th annual meeting of the world muscle society, Oct 18-22 2011, Almancil, Portugal: Development of systemic antisense treatment in dystrophic mouse models for Duchenne Muscular Dystrophy 12. Cold Spring Harbor Laboratory course on RNA Therapeutics, Dec 4-7 2011, Cold Spring Harbor, NY, USA: Development of systemic antisense treatment in dystrophic mouse models for Duchenne muscular dystrophy (invited lecture) 2010 1. Nederlandse Vereniging van Gen Therapie, March 11 2010, Heuze, the Netherlands: Exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 2. Nederlandse Vereniging van Gen Therapie, March 12 2010, Heuze, the Netherlands: Antisensemediated exon skipping as a therapy for Duchenne muscular dystrophy 3. Cold spring harbor meeting RNA and oligonucleotides therapeutics, April 7-10 2010, Cold Spring Harbor, NY, USA: Long term systemic antisense-mediated exon skipping in dystrophic mouse models. 4. Belgium meeting of Child Neurology, April 24 2010, Gent, Belgium: Use of antisense oligonucleotides. 5. International Translational Research Conference for NMDs, June 17-19 2010, Sao Paulo, Brazil: Innovative therapeutic approaches for DMD (invited lecture) 6. EURASNET mRNA and mis-splicing: disease and therapeutics, July 13-15 2010, Cambridge, UK: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 7. 12th international congress on neuromuscular diseases, July 17-22 2010, Naples, Italy: Learning from early trials of antisense oligonucleotide therapy for DMD (invited lecture) 8. NIH/FDA Conference on Antisense Oligonucleotide Therapies in Neuromuscular Disease, September 27 2010, Washington DC, USA: TBA 9. Seminar series of the Nationwide Children’s Hospital, Columbus (OH), USA, September 30 2010: Antisense oligonucleotides treatment for neuromuscular and neurological disorders
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Curriculum vitae
Annemieke Aartsma-Rus
10. European Society of Gene and Cell Therapy, Educational session, October 22 2010, Milan, Italy: Antisense-mediated exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 11. Portuguese Human Genetics Conference, November 18-20 2010, Coimbra, Portugal: Development of antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 2009 1. Keystone meeting on Therapeutic Modulation of RNA Using Oligonucleotides, Feb 8-13 2009, Lake Louise (AB) Canada: Clinical trials for antsisense-mediated exon skipping for Duchenne Muscular Dystrophy (invited lecture) 2. Nederlandse Vereniging van Humane Genetica meeting, 23-24 April 2009, Veldhoven the Netherlands: First clinical trial on therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 3. Meeting Molecular Therapeutics of the Interdisciplinary Centre for Clinical Research (IZKF) of the University of Erlängen (Germany), 14-16 May 2009, Nürnberg Germany: Antisense-mediated exon skipping: preclinical and clinical development of a therapy for Duchenne muscular dystrophy (invited lecture) 4. Lecture at AVI-Biopharma, Corvallis, USA, May 26 2009: Development of antisense-mediated exon skipping for Duchenne muscular dystrophy – from design to clinical trials (invited lecture) 5. 2009 annual meeting of the American Society of Gene Therapy, May 29 2009: Systemic AntisenseMediated Exon Skipping in Mouse Models for Duchenne Muscular Dystrophy 6. Francophone Society of Cell and Gene Therapy Meeting, 21-23 June 2009, Paris France: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 7. Oxford University, July 9 2009, Oxford UK: Development of antisense-mediated exon skipping for Duchenne muscular dystrophy; from design to clinical trials (invited lecture) 8. Workshop on alternative splicing and disease, Montpellier, France, July 20 2009: Antisensemediated exon skipping therapy for Duchenne muscular dystrophy – design, preclinical studies and clinical trials (invited lecture) 9. Cold Spring Harbor Laboratories, August 17 2009, Cold Spring Harbor, NY, USA: Development of antisense-mediated exon skipping therapy for Duchenne muscular dystrophy – from proof of concept to preclinical and clinical trials (invited lecture) 10. EMEA headquarters, September 25 2009. TREAT-NMD workshop “The development of antisense oligonucleotide therapies for Duchenne Muscular Dystrophy”: The specific issues of personalized medicine in Duchenne muscular dystrophy 11. Joint meeting of the oligonucleotide therapeutic society and antisense society Japan, workshop “oligos targeting DMD and diseases involving splicing” 3-6 Nov 2009, Fukuota, Japan; speaker (long term systemic studies with antisense oligonucleotides in Duchenne muscular dystrophy models) and panel member 12. International symposium on DMD exon skipping, Nov 10 2009, Kobe, Japan: Development of exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 13. Eurotides meeting, December 3 2009, Amsterdam, the Netherlands: Preclinical development of systemic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 2005-2008 1. Séminaire Généthon, February 24 2005, Evry, France: Development of an antisense-mediated exon-skipping therapy for Duchenne Muscular Dystrophy (invited lecture) 2. ESF and ICGDB joint workshop on The pathology of pre-mRNA splicing: diagnostic and mechanistic aspects, April 7-9 2005, Trieste, Italy: Blocking SR binding sites by antisense oligonucleotides to indice exon skipping: therapeutic applications for Duchenne Muscular Dystrophy 3. Symposium on Alternate Transcript Diversity II - Biology, and Therapeutics, March 21-23, 2006, Heidelberg, Germany: Therapeutic antisense-mediated exon skipping for Duchenne Muscular Dystrophy (invited lecture) 4. 9th Annual meeting of the American Society of Gene Therapy, May 31 - June 4, 2006, Baltimore, USA: First-in-Man Study on Antisense-Induced Exon Skipping for Duchenne Muscular Dystrophy (invited lecture) 5. 28e congres Kindergeneeskunde (Pediatrics), November 1-3, 2006, Veldhoven, The Netherlands: Antisense-gemedieerde exon skip therapie voor Duchenne Spierdystrofie (invited lecture) 6. Eurostemcell clinical workshop: Towards clinical trials in Duchenne Muscular Dystrophy, April 3-4, 2007, Bellagio, Italy: Towards clinical application of antisense-mediated exon skipping for DMD
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Curriculum vitae
Annemieke Aartsma-Rus
(invited lecture) 7. 7th National Congress, Italian Association for Myology, May 25-26 2007, Ferrara, Italy: The development of antisense-mediated exon skipping therapy for Duchenne muscular dystrophy (invited lecture) 8. 12th annual meeting of the RNA Society, May 28 - June 3 2007, Madison, USA: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy 9. 2007 Annual meeting of the American Society of Human Genetics 23-27 October 2007, San Diego USA: Antisense-mediated exon 51 skipping restores local dystrophin expression in muscle of Duchenne muscular dystrophy patients 10. 15th Meeting of the European Society of Gene and Cell Therapy 27-30 October 2007, Rotterdam The Netherlands: In vivo optimization of therapuetic antisense-mediated exon skipping for Duchenne muscular dystrophy 11. European conference of human genetics meeting 2008, 31 May - 3 June, Barcelona Spain: Systemic antisense-mediated exon skipping studies in mouse models for Duchenne muscular dystrophy 12. First international meeting of the EURASNET consortium, on alternative splicing 21-23 May 2008, Krakaw Poland: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) 13. 13th annual meeting of the RNA Society, July 28 - August 3 2008, Berlin, Germany: First clinical trial on therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy 14. Neurotherapy Summerschool of brain research (Dutch Institute of Neuroscience), August 25-28, Amsterdam, the Netherlands: Therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture; replacing Mimoun Azzouz) 15. Fourth annual meeting of the oligonucleotide therapeutic society, 15-18 October 2008, Boston (MA) USA: Antisense-mediated exon skipping for Duchenne muscular dystrophy (invited lecture) Lectures at Patient/Parents meetings Vereniging Spierziekte Nederland, 2005 and 2006 Dutch Duchenne Parent Project, 2006, 2008, 2009, 2010, 2011, 2013, 2014 and 2015 Action Duchenne (UK), 2005, 2007, 2008, 2011, 2012, 2013 and 2014 Parent Project Muscular Dystrophy, 2009, 2010, 2011 and 2013 Asian United Parent Project (India), 2009 and 2014 Italian Parent Project, 2008, 2010, 2011, 2012, 2013, 2014 and 2015 Brazilian International Conference on NMDs (Patient day) 2010 Webinar for Duchenne Foundation (Australia), 2009, 2010 UPPMD Webinar, 2014 Australian Duchenne Foundation meeting 2012 Rumanian parent project 2011 Chinese Dear Mommy and Daddy (DMD) conference for DMD patients and parents 2011 and 2013 Swedish Parent Project 2012 and 2014 I have also given educational lectures to neurologists in India (2009), Brazil (2010), China (2011), Rumania (2011), Denmark (2011) and Sweden (2012 and 2014) and during a Masterclass for European neurologists (2015) Other Relevant Information
2015 Selected as the most influential scientist in Duchenne muscular dystrophin in the past 10 years (since 2005) by Expertscape based on contributions to the understanding and treatment of Duchenne muscular dystrophy (http://expertscape.com/ex/muscular+dystrophy%2C+duchenne) 2014 Listed on the 2014 VIVA-400 list containing the 400 most inspiring Dutch women (http://viva400.viva.nl/knappe-koppen/annemieke-aartsma-rus/); voted in the top 5 in the category “brainics” by public votes 2014 Selected as the second most influential scientist in Duchenne muscular dystrophy in the past 10 years (2005-2014) by Expertscape based on contributions to the understanding and treatment of Duchenne muscular dystrophy (http://www.prlog.org/12285052-expertscape-ranks-worlds-topdoctors-and-hospitals-in-duchenne-muscular-dystrophy.html 2014 Selected as a member (2014-18) of the junior section of the Dutch Royal Academy of Sciences
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Curriculum vitae
Annemieke Aartsma-Rus
(consisting of the top 50 young (
