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Article

AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.

Trends in molecular medicine - 1 Jun 2021

Yang Huiya, Brown Robert H, Wang Dan, Strauss Kevin A, Gao Guangping

Abstract excerpt

De novo glycosphingolipid (GSL) biosynthesis defects cause severe neurological diseases, including hereditary sensory and autonomic neuropathy type 1A (HSAN1A), GM3 synthase deficiency, and hereditary spastic paraplegia type 26 (HSPG26), each lacking effective treatment. Recombinant adeno-associated virus (AAV)-mediated gene therapy has emerged as a powerful treatment for monogenic diseases and might be...

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Identifiers

PMID: 33714697
DOI: 10.1016/j.molmed.2021.02.004
PMCID: PMC8840833

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