The American Society of Gene and Cell Therapy has a great overview called Gene Therapy 101.

This document was also provided to us to share with our community by David Barrett, Executive Director of the American Society of Gene and Cell Therapy. Click on the image to view an Introduction to Gene and Cell Therapy. By learning more about gene therapy overall, it will be possible to better understand GM1 Gangliosidosis gene therapy programs.

GM1 Gangliosidosis Gene Therapy Programs

At this time, there are four companies with GM1 Gangliosidosis AAV gene therapy programs. However, none of the clinical trials are currently recruiting. To learn more about those programs, please see this page.