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

• Principais autores: Alex Mas Monteys (Autor), Matthew J Wilson (Autor), Ryan L Boudreau (Autor), Ryan M Spengler (Autor), Beverly L Davidson (Autor)
• Formato: Livro
• Publicado em: Elsevier, 2015-01-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: 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...