DMD Genetic Therapy Group

Welcomeat the website of the DMD Genetic Therapy Group

The primary mission of the DMD Genetic Therapy Group at the Department of Human Genetics (chaired by Prof. Dr. Silvre van der Maarel) 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 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 Prosensa are developing antisense oligonucleotides (AONs) as small synthetic molecule drugs for DMD.

The Department of Human Genetics has an excellent track record in the field of duchenne muscular dystrophy. It set up the first DNA based carrier detection and prenatal diagnosis worldwide, performed etiological DNA, RNA and protein research, and developed diagnostics, for DMD and other muscular diseases, in an international framework.

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.

In 2007, the first clinical trial on antisense-mediated exon skipping was successfully completed by the LUMC and Prosensa.For more updated news about the group and the different research projects; please go the the news page.

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 an easier to understand therms and images. 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 Exonskipping and the DMD disease. Please have a look!

Here you will find more background information about the disease.

Here you will find all the scientific publications about Duchenne.

Hereyou will find more background information about the Exon skipping technology

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DMD Genetic Therapy Group