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 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.

For Parents of Duchenne Muscular Dystrophy patients we have created a special website, written in a non-scientific language. This website is available in Dutch and English.

A 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!

What’s hot in the research field?

Information on new developments in the genetic therapy field

Technology & development

Background information about exon skipping and biomarkers  

Applicability of exon skipping

Exon skipping for deletions, duplications or point mutations

Do you have a question about DMD or do you want to get in contact with Prof. dr. Annemieke Aartsma-Rus? Please leave a comment on the contact page.