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AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.

Authors :
Yang, Huiya
Brown, Robert H.
Wang, Dan
Strauss, Kevin A.
Gao, Guangping
Source :
Trends in Molecular Medicine. Jun2021, Vol. 27 Issue 6, p520-523. 4p.
Publication Year :
2021

Abstract

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. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
14714914
Volume :
27
Issue :
6
Database :
Academic Search Index
Journal :
Trends in Molecular Medicine
Publication Type :
Academic Journal
Accession number :
150337105
Full Text :
https://doi.org/10.1016/j.molmed.2021.02.004