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

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

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 parti...

Bibliografske podrobnosti
Main Authors:	Scholefield, J, Greenberg, L, Weinberg, MS, Arbuthnot, P, Abdelgany, A, Wood, M
Format:	Journal article
Jezik:	English
Izdano:	2009

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