Scientific Board of Advisory

Dr. Dongsheng Duan - Scientific Advisory Board Member

See Dr. Duan's profile on University of Missouri here. 

Dr. Duan is a Curators’ Distinguished Professor and Margaret Proctor Mulligan
Professor in Medical Research at the University of Missouri, School of Medicine,
Department of Molecular Microbiology and Immunology.

Dr. Duan is a fellow of the National Academy of Inventors and a fellow of the American Association for the Advancement of Science (AAAS).

Dr. Duan received his Ph.D. in 1997 from the University of Pennsylvania. His research focuses on the development of novel AAV
vectors and gene therapy for Duchenne muscular dystrophy in small and large animal models.

He is the recipient of the American Society of Gene Therapy Outstanding New
Investigator Award in 2006.

He also received Spurgeon Distinguished Medical Research Award in 2004, Chancellor’s Award for Outstanding Research and Creative Activity at the University of Missouri in 2009, Honorary Medical Alumni Award in 2017, and Excellence in Research Mentoring of Trainees Award in 2017.

Dr. Duan has published more than 200 research articles and edited/co-edited three muscle gene therapy books.

His papers have been cited more than 14,181 times (H index: 66; i10
index: 161).

He serves as an associate editor for Human Gene Therapy, Molecular Therapy, Frontiers in Physiology, and Frontiers in Genome Editing.

Dr. Annemieke Aartsma-Rus - Scientific Advisory Board Member

See Dr. Aartsma-Rus profile in Treat-NMD here. 

Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center (LUMC, the Netherlands). She played an important role in the development of antisense mediated exon skipping for Duchenne muscular dystrophy during her PhD research (2000-2004) at the LUMC. She is a PI since 2007. Since 2013 she has a visiting professorship at the John Walton Muscular Dystrophy Research Center of Newcastle University (UK). In 2020 she co-founded the Dutch Center for RNA Therapeutics (DCRT), a non-for-profit academic collaboration aiming to develop clinical treatment with exon skipping therapies for eligible patients with unique mutations. In 2022 she became a board member of the N-of-1 collaborative (N1C), a global umbrella organization aiming to facilitate development of antisense oligonucleotide therapies for patients with very rare, eligible mutations.

Her work currently focuses on developing antisense-mediated exon skipping as a therapy for Duchenne muscular dystrophy and rare brain diseases. This involves work in cell and animal models to improve efficiency of exon skipping, studies in muscle pathology, the identification of biomarkers, studying the basics of pre-mRNA splicing and transcript processing and the generation and detailed analysis of mouse models. Finally, she aims to bridge the gap between stakeholders (patients, academics, regulators and industry) involved in drug development for rare diseases and to develop exon skipping therapies for patients with unique mutations.

She has published over 220 peer-reviewed papers, 11 book chapters and 15 patents. She has given many invited lectures at scientific conferences and patient organization meetings, where she is known for her ability to present science in a clear and understandable way. She created and maintains an overview of different therapeutic approaches for Duchenne on the TREAT-NMD website (https://treat-nmd.org/research-overview/dmd-research-overview/).

In 2011 she received the Duchenne Award from the Dutch Duchenne Parent Project in recognition of her dedication to the Duchenne field. In 2020 she received the Black Pearl Science Award from Eurordis for her work in educating patients. In 2021 she received the Ammodo Science Award for her contribution to developing exon skipping therapies for Duchenne, the outstanding achievement award from the Dutch Society of Gene and Cell Therapy for her work and the Rosalind Franklin in Science award for her work for the journal Nucleic Acid Therapeutics. Since 2015 she is the most influential scientist in Duchenne muscular dystrophy in the past 10 years (https://expertscape.com/ex/muscular+dystrophy%2C+duchenne).

She is chair of the TREAT-NMD Advisory Committee for Therapeutics (TACT), Chair of the Dutch Center for RNA Therapeutics, vice-chair of COST Action CA17103 (Delivery of antisense RNA therapies), board member of the N-of-1 Collaborative, was President of the Oligonucleotide Therapeutics Society (2019-2021), and was Chair of the TREAT-NMD executive committee (2013-2016 and 2019-2020). She was a junior member of the Dutch Royal Academy of Sciences (DJA), consisting of the top 50 scientists in the Netherlands under 45, from 2014-2019. She is part of the core group of the N-of-1 collaborative and 1 mutation 1 medicine (1M1M) network.

She has successfully applied for numerous grant applications, including a VIDI award (€800,000) from the Dutch government (ZonMw) in 2009 and collaborative grants in 2012 (Rare Disease Program, €3,000,000) and 2021 (PSIDER, €4,000,000). She is/was involved in multiple EU projects, e.g. TREAT-NMD (FP6), Bio-NMD (FP7), NeurOmics (FP7), BIND (Horizon2020) and COST Actions BM1208 and CA17103.

She has been instrumental in setting up training courses, e.g. TREAT-NMD Duchenne masterclasses, the TREAT-NMD/EURO-NMD translational summer school and a COST training school on oligonucleotide therapy development (2020). She is a member of the Therapies Scientific Committee of the International Rare Disease Research Consortium (IRDiRC). 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 and Therapeutic Advances in Rare Disease.