Article

AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial

2025-07-29

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> GM1 gangliosidosis, caused by biallelic variants in GLB1 , results from deficiency of lysosomal β-galactosidase, the enzyme primarily responsible for degradation of GM1 ganglioside. This progressive neurodegenerative disease is uniformly fatal with no approved therapies, but preclinical studies utilizing gene therapy have shown promising results. <h4>Methods</h4> This pha...

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Literature Corpus work: 759691d8-749d-510a-9c2c-e1feb3f7bf76
DOI: 10.1101/2025.07.28.25332074

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