Jessie's Rally of Hope
GM1 is a lysosomal storage disorder. It is caused by a mutation in the gene responsible for a vital enzyme called betagalactosidase (β-gal). The role of β-gal is to degrade a fatty substance or lipid called GM-1 ganglioside. In the absence of β-gal, GM-1 accumulates abnormally in cells, especially in the nerve cells, or neurons, of the brain. This ongoing accumulation, or "storage", of GM-1 causes progressive damage and eventually death of the cells.
There are several projects currently focused on GM1 from natural history studies to gene therapy. Stay tuned for future updates on the website.
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