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AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
- 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