The DMD Genetic Therapy Group
The
primary mission of the DMD Genetic Therapy Group
at the Department of
Human Genetics (chaired by Prof. Dr. Gert-Jan B. van Ommen) of
the
Leiden University
Medical Center is the development of the "exon skipping"
approach 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
the exon skip approach
for DMD.
We have created a special section on our website for patients and parents, where we try to explain the rationale of the exon skipping approach in simple terms. If anything is unclear, please do not hesitate to contact us for further information. This will help us to improve these pages.