AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
Ontology highlight
ABSTRACT:
SUBMITTER: Yang H
PROVIDER: S-EPMC8840833 | biostudies-literature | 2021 Jun
REPOSITORIES: biostudies-literature
Publications
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
Yang Huiya H Brown Robert H RH Wang Dan D Strauss Kevin A KA Gao Guangping G
Trends in molecular medicine 20210310 6
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 particularly suitable for these neurological conditions. ...[more]
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