Article

Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study

2022-04-17

Abstract excerpt

Ganglioside-monosialic acid (GM1) gangliosidosis, a rare autosomal recessive disorder, is frequently caused by deleterious single nucleotide variants (SNVs) in GLB1 gene. These variants result in reduced β-galactosidase (β-gal) activity, leading to neurodegeneration associated with premature death. Currently, no effective therapy for GM1 gangliosidosis is available. Three ongoing clinical trials aim to deliver a...

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Literature Corpus work: 716e38a4-e676-50bc-a4bb-979a8e74808b
DOI: 10.1101/2022.04.17.488473

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