AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. Issue 6 (June 2021)

• Record Type: Journal Article
• Authors: Yang, Huiya; Brown, Robert H.; Wang, Dan; Strauss, Kevin A.; Gao, Guangping
• Is Part Of: Trends in molecular medicine. Volume 27:Issue 6(2021)
• Page Start: 520
• Page End: 523
• Publication Date: 2021-06
• Subjects: adeno-associated virus -- gene therapy -- glycosphingolipid biosynthesis deficiency; Molecular biology -- Periodicals; Pathology, Molecular -- Periodicals; Physiology, Pathological -- Periodicals; 572.8
• DOI: 10.1016/j.molmed.2021.02.004
• Languages: English
• ISSNs: 1471-4914

Abstract

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.