
Two Roads to the Same Goal: AAV Gene Therapy vs. Ex Vivo Lentiviral Gene Therapy for GM1
A plain-language guide for families: what these two approaches are, how they differ, and what a new research paper adds to the picture.
In partnership with Cure GM1 Foundation
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A plain-language guide for families: what these two approaches are, how they differ, and what a new research paper adds to the picture.

GM1 and GM2 are close cousins, but they are distinct diseases caused by different genes and different missing enzymes. Where the two actually diverge.

From the first clinical descriptions to modern therapeutics — nearly a century in which GM1 was read as Tay-Sachs, until biochemistry told the two apart.
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