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Genes 2016, 7(12), 132; doi:10.3390/genes7120132

Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells

Department of Molecular Biomedicine, Institute of Bioorganic Chemistry, Polish Academy of Sciences, Noskowskiego 12/14 Str., 61-704 Poznan, Poland
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Authors to whom correspondence should be addressed.
Academic Editor: Wenyi Gu
Received: 29 September 2016 / Revised: 6 December 2016 / Accepted: 9 December 2016 / Published: 17 December 2016
(This article belongs to the Special Issue RNA Interference 2016)
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Abstract

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 polyQ diseases. Herein, we demonstrate that the selective targeting of a repeat tract in a mutant ATXN7 transcript by RNA interference is a feasible approach and results in an efficient decrease of mutant ataxin-7 protein in patient-derived cells. Oligonucleotides (ONs) containing specific base substitutions cause the downregulation of the ATXN7 mutant allele together with the upregulation of its normal allele. The A2 ON shows high allele selectivity at a broad range of concentrations and also restores UCHL1 expression, which is downregulated in SCA7. View Full-Text
Keywords: spinocerebellar ataxia type 7; siRNA; CAG repeats; polyglutamine diseases; allele-selective silencing spinocerebellar ataxia type 7; siRNA; CAG repeats; polyglutamine diseases; allele-selective silencing
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Fiszer, A.; Wroblewska, J.P.; Nowak, B.M.; Krzyzosiak, W.J. Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells. Genes 2016, 7, 132.

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