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

• Main Authors: Edith L. Pfister (Author), Kathryn O. Chase (Author), Huaming Sun (Author), Lori A. Kennington (Author), Faith Conroy (Author), Emily Johnson (Author), Rachael Miller (Author), Florie Borel (Author), Neil Aronin (Author), Christian Mueller (Author)
• Format: Book
• Published: Elsevier, 2017-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 tole...