Department of Human Genetics | Leiden University Medical Center | Leiden, The Netherlands

The primary mission of the DMD Genetic Therapy Group at the Department of Human Genetics of the Leiden University Medical Center  is the development of a genetic therapy for Duchenne muscular dystrophy (DMD).

The group started in 1998 and is since February 2007 supervised and coordinated by Prof. dr. Annemieke Aartsma-Rus, after Dr. Judith C.T. van Deutekom left to work as Head of Research for the biotech company Prosensa (currently BioMarin, the Netherlands). In a joint effort the DMD Genetic Therapy Group and BioMarin are developing antisense oligonucleotides (AONs) as small synthetic molecule drugs for DMD.

The DMD Genetic Therapy group studies muscle pathology in dystrophic cell and animal model systems, ways to improve muscle quality, the identification and validation of biomarkers and the processing of the dystrophin transcripts. The group was awarded for its research by the Princess Beatrix Fund in 2001 and by the LUMC in 2003 (C.J. Kok Award).

The group consists of 1 Principal Investigator, 2 Assistent Professors, 2 Postdoctoral Researchers, 5 Ph.D. students and 5 Research Technicians. To learn more about our group members, click here.

Exon skipping | DMD Genetic Therapy group Leiden