Welcome to the website of the DMD Genetic Therapy group Leiden

The DMD Genetic Therapy group

Welcome to the website of the DMD Genetic Therapy group. Our group started in 1998 and is supervised and coordinated by Prof. dr. Annemieke Aartsma-Rus since February 2007. The primary mission of our group is the development of a genetic therapy for Duchenne muscular dystrophy (DMD), with focus on 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. On this website we want to inform you about the scientific background of DMD and the concept of exon skipping. We hope you can find all the information you are looking for, otherwise do not hesitate to contact us.

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

Applicability of exon skipping

Exon skipping for deletions, duplications or point mutations

DMD Genetic Therapy group Leiden

News from our group and information about our group members

Technology & development

Information about exon skipping and biomarkers

For Parents of Duchenne muscular dystrophy patients we have created a special website, written in a non-scientific language. This website can be found under INFORMATION FOR PARENTS (available in Dutch and English).

This video has been created to show a “behind the scenes footage” of our work in Leiden and tells a bit more about exon skipping and Duchenne muscular dystrophy.