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

• Main Authors: Fabiola V. Borbolla-Jiménez (Author), María Luisa Del Prado-Audelo (Author), Bulmaro Cisneros (Author), Isaac H. Caballero-Florán (Author), Gerardo Leyva-Gómez (Author), Jonathan J. Magaña (Author)
• Format: Book
• Published: MDPI AG, 2021-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...