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Article

Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype.

PloS one - 30 Sept 2009

Scholefield Janine, Greenberg L Jacquie, Weinberg Marc S, Arbuthnot Patrick B, Abdelgany Amr, Wood Matthew J A

Abstract excerpt

Spinocerebellar ataxia type 7 is a polyglutamine disorder caused by an expanded CAG repeat mutation that results in neurodegeneration. Since no treatment exists for this chronic disease, novel therapies such post-transcriptional RNA interference-based gene silencing are under investigation, in particular those that might enable constitutive and tissue-specific silencing, such as expressed hairpins. Given that...

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Identifiers

PMID: 19789634
DOI: 10.1371/journal.pone.0007232
PMCID: PMC2747278

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