Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo

• Main Authors: Alex Mas Monteys (Author), Matthew J Wilson (Author), Ryan L Boudreau (Author), Ryan M Spengler (Author), Beverly L Davidson (Author)
• Format: Book
• Published: Elsevier, 2015-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Huntington's disease (HD) is a dominantly inherited neurodegenerative disease caused by CAG repeat expansion in exon 1 of huntingtin (HTT). Studies in mouse models of HD with a regulated mutant transgene show that continuous mutant allele expression is required for behavioral and pathological s...