Learn About Gene Therapy for Pompe Disease

ASGCT Staff - November 19, 2019

Pompe disease occurs when the genes that instruct our cells to create the GAA enzyme are faulty. The GAA enzyme’s job is to break down complex molecules like glycogen, which is a substance that helps our body store energy.

The American Society of Gene & Cell Therapy (ASGCT) released its latest resource profiling disease-specific treatment efforts, this time focusing on developing gene therapies for Pompe disease.

Watch ASGCT's Pompe Disease and Gene Therapy Video

The resources on Pompe disease are now available on Pompe disease is a genetic disorder that occurs when our bodies lack an important digestive enzyme called acid alpha-glucosidase, or GAA for short. You can learn more about the effect of Pompe disease on the body here.

The information on Pompe disease is part of the launch of a second round of material in ASGCT’s Patient Education program. ASGCT has been releasing new resources weekly on the Patient Education portal. Check out what we have released so far, and keep an eye on our website for the last  new resource of 2019—CAR T Basics—on December 3.

Let us know what you think of this second batch of resources. Send feedback via the ASGCT Contact form or at

Share This Page

Related Articles


Watch “Understanding the Gene Therapy Process and Aftercare,” from ASGCT and NORD

ASGCT Staff - December 06, 2019

Learn About Blood Cancers and CAR T-Cell Therapy

ASGCT Staff - December 03, 2019

Watch “The FDA's Role in Gene Therapy,” from ASGCT and NORD

ASGCT Staff - November 15, 2019

Learn About Gene Therapy for Hemophilia

ASGCT Staff - November 12, 2019