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Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells

Spinocerebellar ataxia type 7 (SCA7) is a human neurodegenerative polyglutamine (polyQ) disease caused by a CAG repeat expansion in the open reading frame of the ATXN7 gene. The allele-selective silencing of mutant transcripts using a repeat-targeting strategy has previously been used for several po...

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Detalles Bibliográficos
Autores principales: Fiszer, Agnieszka, Wroblewska, Joanna P., Nowak, Bartosz M., Krzyzosiak, Wlodzimierz J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5192508/
https://www.ncbi.nlm.nih.gov/pubmed/27999335
http://dx.doi.org/10.3390/genes7120132