Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. She played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy during her PhD research (2000-2004) at the Leiden University Medical Center (the Netherlands). As of December 2007 she became leader of the “DMD exon skip group”. Since 2013 she has a visiting professorship at the Institute of Genetic Medicine of Newcastle University (UK).
She is President of the Oligonucleotide Therapeutics Society (2019-2020) and vice-chair of COST Action CA17103 (Delivery of antisense RNA therapies) and the TREAT-NMD executive committee. In 2013 she was elected a member of the junior section of the Dutch Royal Academy of Sciences (DJA), which consists of what are considered the top 50 scientists in the Netherlands under 45. She has been selected as most influential scientist in Duchenne muscular dystrophy in the past 10 years by Expertscape based on contributions to the understanding and treatment of Duchenne muscular dystrophy four times in a row (2015-now).
Thus far, she has published over 180 peer-reviewed papers and 11 book chapters, as well as 15 patents and has edited one book. She has given many invited lectures at meetings, symposia and workshops as well as patient/parent organizations meetings, where she is known for her ability to present science in a clear and understandable way. She has created and maintains multiple websites on therapeutic approaches for aimed at patients and parents. In 2011 she received the Duchenne Award from the Dutch Duchenne Parent Project in recognition of this work and her dedication to the Duchenne field. She also writes regular blogs for the LUMC.
She has successfully applied for numerous grant applications, including a prestigious VIDI award (€800,000) from the Dutch government in 2009. She is/was involved in multiple EU projects, e.g. TREAT-NMD (FP6), Bio-NMD (FP7), NeurOmics (FP7) and COST Action CA17103. She is a member of the Therapies Scientific Committee of the International Rare Disease Research Consortium (IRDiRC). She was chair of the COST Action “Networking towards clinical application of antisense-mediated exon skipping for rare diseases” (2013-2017), and Chair of the executive board of the TREAT-NMD alliance (2014-2016). She is co-editor in chief of Nucleic Acid Therapeutics, and serves on multiple editorial boards, e.g. Journal of Neuromuscular Diseases (associate editor), Molecular Therapy, Therapeutic Advances in Rare Disease and Cardiovascular Genetics and Genomics. She is a member of the TREAT-NMD project ethics counsel and of the TREAT-NMD Advisory Committee for Therapeutics (TACT) and the scientific advisory board of Genethon.
Department of Human Genetics
Leiden University Medical Center
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