Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin

Huntington’s disease is a devastating, incurable neurodegenerative disease affecting up to 12 per 100,000 patients worldwide. The disease is caused by a mutation in the Huntingtin (Htt) gene. There is interest in reducing mutant Huntingtin by targeting it at the mRNA level, but the maximum tolerable...

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Pubblicato in:Mol Ther Nucleic Acids
Autori principali: Pfister, Edith L., Chase, Kathryn O., Sun, Huaming, Kennington, Lori A., Conroy, Faith, Johnson, Emily, Miller, Rachael, Borel, Florie, Aronin, Neil, Mueller, Christian
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5424568/
https://ncbi.nlm.nih.gov/pubmed/28624208
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.04.011
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