The DMD Genetic Therapy Group

The primary mission of the DMD Genetic Therapy Group at the Department of Human Genetics (chaired by Prof. Dr. Silvère van der Maarel) of the Leiden University Medical Center (Division 4) 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 (see "news").



Dr. Annemieke Aartsma-Rus

DMD Genetic Therapy Group

Dept. of Human Genetics,

Leiden University Medical Center, Div. 5

Postzone S4-P

PO Box 9600

2300 RC Leiden

The Netherlands

Tel. +31-71-5269436

Fax +31-71-5268285

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