New Perspectives of Gene Therapy on Polyglutamine Spinocerebellar Ataxias: From Molecular Targets to Novel Nanovectors

New Perspectives of Gene Therapy on Polyglutamine Spinocerebellar Ataxias: From Molecular Targets to Novel Nanovectors

Seven of the most frequent spinocerebellar ataxias (SCAs) are caused by a pathological expansion of a cytosine, adenine and guanine (CAG) trinucleotide repeat located in exonic regions of unrelated genes, which in turn leads to the synthesis of polyglutamine (polyQ) proteins. PolyQ proteins are pron...

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Bibliographic Details
Main Authors: Fabiola V. Borbolla-Jiménez, María Luisa Del Prado-Audelo, Bulmaro Cisneros, Isaac H. Caballero-Florán, Gerardo Leyva-Gómez, Jonathan J. Magaña
Format: Article
Language:English
Published: MDPI AG 2021-07-01
Series:Pharmaceutics
Subjects:
spinocerebellar ataxias
polyglutamine
gene therapy
expanded triplet repeat
antisense and interferent technology
DNA editing systems
Online Access:https://www.mdpi.com/1999-4923/13/7/1018

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https://www.mdpi.com/1999-4923/13/7/1018

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