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

Title
Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin
Authors
Keywords
Huntington’s disease, gene therapy, RNAi, AAV, miRNA, shRNA, huntingtin, HTT
Journal
Molecular Therapy-Nucleic Acids
Volume 7, Issue -, Pages 324-334
Publisher
Elsevier BV
Online
2017-04-15
DOI
10.1016/j.omtn.2017.04.011

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