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...

Celý popis

Podrobná bibliografie

Hlavní autoři:	Fabiola V. Borbolla-Jiménez, María Luisa Del Prado-Audelo, Bulmaro Cisneros, Isaac H. Caballero-Florán, Gerardo Leyva-Gómez, Jonathan J. Magaña
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	MDPI AG 2021-07-01
Edice:	Pharmaceutics
Témata:	spinocerebellar ataxias polyglutamine gene therapy expanded triplet repeat antisense and interferent technology DNA editing systems
On-line přístup:	https://www.mdpi.com/1999-4923/13/7/1018
Tagy:	Přidat tag Žádné tagy, Buďte první, kdo vytvoří štítek k tomuto záznamu!