Gene therapy for muscle disease

Project summary

Gene therapy is the collective name of therapies, in which the genetic material of a patient, the DNA, is intervened. Gene therapy could be a good treatment for hereditary muscle diseases. These muscle diseases are caused by a defective gene: a genetic error that leads to the disease. In about 80% of all muscle diseases, the error is in the DNA.

Three research teams in Leiden, Nijmegen and Rotterdam can start their research. They will use various techniques to further develop gene therapy for muscle diseases. Spierfonds Policy & Research Manager Dr. Ellen Sterrenburg explains: “We support various lines of gene therapy, across the board. It is really an ambitious program. But we believe it is possible. The Dutch researchers are at the top internationally.

The team in Leiden, led by Prof. Dr. Niels Geijsen, will focus their research on Duchenne’s disease. The team in Nijmegen, led by Prof. Hans van Bokhoven, focuses on myotonic dystrophy. And the team in Rotterdam, led by Dr. Pim Pijnappel, is conducting research into gene therapy for Pompe disease. The three research teams use different approaches in their research. These may also be applicable to other muscle diseases in the future.

The goal is to test gene therapy in patients within five years.