Gene therapy for Pompe disease effective in mice

Single dose of gene therapy could augment or replace frequent enzyme infusions

Date:

January 26, 2017

Source:

Duke University Medical Center

Summary:

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

Author: Ashok Vellodi

I have had a lifelong interest in lysosomal storage disorders. Having recently retired I now have time to pursue my passion for teaching and training of young doctors and scientists, and hopefully instil in them the passion for their patients that is so integral to working in this field. View all posts by Ashok Vellodi