Welcome to the website of the DMD Genetic Therapy Group
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 project started in 1998 and is currently supervised by Prof. Dr. Annemieke Aartsma-Rus, after Dr. Judith C.T. van Deutekom left in February 2007 to work as Head of research for the biotech company Prosensa B.V. (Leiden, the Netherlands). In a joint effort the DMD Genetic Therapy Group and BioMarin (previously Prosensa) are developing antisense oligonucleotides (AONs) as small synthetic molecule drugs for DMD.
The DMD Genetic Therapy group also 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 DMD Genetic Therapy Group was awarded for its research by the Princess Beatrix Fund in 2001 and by the LUMC (C.J. Kok Award) in 2003.
We hope you find all the information you are looking for on this webpage. If not please do not hesitate to contact us by using the contact form.
For Parents of Duchenne Muscular Dystrophy patients we have created a special website (in Dutch and English), written in a non-scientific language. Please have a look and let us know if you miss information.
Recently a new video has been created to show a “bit more behind the scene footage” of our work here in Leiden and tells a bit more about exon skipping and the DMD disease.
Please have a look!
Patient friendly pages
Information on new developments in the genetic therapy field
Background information about the exon skipping technology