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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...

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Detalles Bibliográficos
Autores principales:	Scholefield, Janine, Greenberg, L. Jacquie, Weinberg, Marc S., Arbuthnot, Patrick B., Abdelgany, Amr, Wood, Matthew J. A.
Formato:	Texto
Lenguaje:	English
Publicado:	Public Library of Science 2009
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2747278/ https://www.ncbi.nlm.nih.gov/pubmed/19789634 http://dx.doi.org/10.1371/journal.pone.0007232

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