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Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway

Huntington’s disease is an incurable neurodegenerative disorder caused by expansion of a CAG trinucleotide repeat within one allele of the huntingtin (HTT) gene. Agents that block expression of mutant HTT and preserve expression of wild-type HTT target the cause of the disease and are an alternative...

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Detalhes bibliográficos
Main Authors: Hu, Jiaxin, Liu, Jing, Yu, Dongbo, Chu, Yongjun, Corey, David R.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3526262/
https://ncbi.nlm.nih.gov/pubmed/23042244
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gks907
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