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

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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Bibliographic Details
Main Authors: Edith L. Pfister, Kathryn O. Chase, Huaming Sun, Lori A. Kennington, Faith Conroy, Emily Johnson, Rachael Miller, Florie Borel, Neil Aronin, Christian Mueller
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
Huntington’s disease
gene therapy
RNAi
AAV
miRNA
shRNA
huntingtin
HTT
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301610

Internet

http://www.sciencedirect.com/science/article/pii/S2162253117301610

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