Single dose of gene therapy could augment or replace frequent enzyme infusions
- January 26, 2017
- Duke University Medical Center
- After decades investigating a rare, life-threatening condition that cripples the muscles, researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients. The therapy uses a modified virus to deliver a gene to the liver where it produces GAA, an enzyme missing in people with Pompe disease.
- Here is the link to the published article
- Low-dose liver targeted gene therapy for Pompe disease enhances therapeutic efficacy of ERT via immune tolerance induction