Gene Therapy webinar series: Episode 6 – Genetic therapies and therapy developments for muscular dystrophies (DMD, LGMD)

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…TIMESLOT: Wednesday 29 May 2024, 16:00 – 17:00 CET

…THEME: Genetic therapies and therapy developments for muscular dystrophies (DMD, LGMD)

…PRESENTERS: Prof. Dr. Annemieke Aartsma-Rus (Leiden University Medical Center, the Netherlands)

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. 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.

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