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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...
Autores principales: | Fiszer, Agnieszka, Wroblewska, Joanna P., Nowak, Bartosz M., Krzyzosiak, Wlodzimierz J. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2016
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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 |
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