Ontology highlight
ABSTRACT:
SUBMITTER: Hu J
PROVIDER: S-EPMC3908448 | biostudies-literature | 2009 Sep
REPOSITORIES: biostudies-literature
Hu Jiaxin J Matsui Masayuki M Corey David R DR
Annals of the New York Academy of Sciences 20090901
The ability to inhibit expression of a mutant allele while retaining expression of a wild-type protein might provide a useful approach to treating Huntington's Disease (HD) and other inherited pathologies. The mutant form of huntingtin (HTT), the protein responsible for HD, is encoded by an mRNA containing an expanded CAG repeat. We demonstrate that peptide nucleic acid conjugates and locked nucleic acids complementary to the CAG repeat selectively block expression of mutant HTT. The selectivity ...[more]