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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Shranjeno v:
Bibliografske podrobnosti
izdano v:Pharmaceutics
Main Authors: Borbolla-Jiménez, Fabiola V., Del Prado-Audelo, María Luisa, Cisneros, Bulmaro, Caballero-Florán, Isaac H., Leyva-Gómez, Gerardo, Magaña, Jonathan J.
Format: Artigo
Jezik:Inglês
Izdano: MDPI 2021
Teme:
Review
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC8309146/
https://ncbi.nlm.nih.gov/pubmed/34371710
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/pharmaceutics13071018
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