Learn About Gene Therapy for GM1 Gangliosidosis

GM1 gangliosidosis (GM1) is a rare genetic disorder caused by a mutation to the GLB1 gene, which instructs cells to produce an enzyme that breaks down fats and sugars in the lysosomes. Without enough of that enzyme, waste builds up in cells and tissues and becomes toxic. Gene therapy for GM1 aims to slow or stop disease progression by delivering a working copy of the GLB1 gene into cells using a viral vector.

Watch this new video to learn more:

On our Patient Education site, you'll also find a new infographic and media kit on GM1 that you can download and share with those in your network who may benefit. Let us know what you think of these resources by sending feedback to ASGCT Patient Outreach Manager Ali Kujawski.

Coming soon: New resources on GM2 (Tay-Sachs and Sandhoff disease)!

Learn About GM1 Gangliosidosis

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