17 June 2017, Vertaalslagen van Fundamenteel naar Toegepast Onderzoek
Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. Since 2013 she has a visiting professorship at the Institute of Genetic Medicine of Newcastle University (UK).
Her work currently focuses on developing antisense-mediated exon skipping as a therapy for Duchenne muscular dystrophy. This involves work in cell and animal models to improve delivery and efficiency of exon skipping compounds, studies in muscle regeneration and pathology, the identification of biomarkers, studying the basics of pre-mRNA splicing and transcript processing and the generation of mouse models. In addition, in collaborative efforts she aims to bridge the gap between different stakeholders (patients, academics, regulators and industry) involved in drug development for rare diseases.
She is the incoming President of the Oligonucleotide Therapeutics Society (2019-2020) and vice-chair of COST Action CA17103 (Delivery of antisense RNA therapies). In 2013 she was elected a member of the junior section of the Dutch Royal Academy of Sciences (KNAW), which consists of what are considered the top 50 scientists in the Netherlands under 45. In 2015, 2016 and 2017 she was selected as most influential scientist in Duchenne muscular dystrophy in the past 10 years by Expertscape.
Thus far, she has published over 160 peer-reviewed papers and 10 book chapters, as well as 15 patents and has edited one book. She has given many invited lectures at scientific and patient meetings. 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 has successfully applied for numerous grant applications, totalling >€10,000,000 as an applicant and >€5,000,000 as a co-applicant. She is co-editor in chief of Nucleic Acid Therapeutics and associate editor for Journal of Neuromuscular Diseases.