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Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7

We evaluate a knockdown-replacement strategy mediated by mirtrons as an alternative to allele-specific silencing using spinocerebellar ataxia 7 (SCA7) as a model. Mirtrons are introns that form pre-microRNA hairpins after splicing, producing RNAi effectors not processed by Drosha. Mirtron mimics may...

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Detalles Bibliográficos
Autores principales:	Curtis, Helen J., Seow, Yiqi, Wood, Matthew J.A., Varela, Miguel A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2017
Materias:	Molecular Biology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5569705/ https://www.ncbi.nlm.nih.gov/pubmed/28575281 http://dx.doi.org/10.1093/nar/gkx483

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