POMPE-CURE: Development of an innovative gene therapy platform for Pompe disease



Pompe disease or glycogen storage disease type II is a potentially life-threatening rare genetic muscle disorder that affects one in 40,000 births. The current POMPE-CURE project aims at developing the next-generation gene therapy to treat Pompe disease. A clinically-relevant Pompe mouse model will be used to assess tissue-specificity, therapeutic efficacy as well as safety of this novel gene therapy platform to ultimately justify a phase I clinical trial in Pompe patients.
Effectieve start/einddatum25/11/2010/11/21