Ontology highlight
ABSTRACT:
SUBMITTER: Hu J
PROVIDER: S-EPMC3908450 | biostudies-literature | 2011 Apr
REPOSITORIES: biostudies-literature
Hu Jiaxin J Gagnon Keith T KT Liu Jing J Watts Jonathan K JK Syeda-Nawaz Jeja J Bennett C Frank CF Swayze Eric E EE Randolph John J Chattopadhyaya Jyoti J Corey David R DR
Biological chemistry 20110207 4
Spinocerebellar ataxia-3 (also known as Machado-Joseph disease) is an incurable neurodegenerative disorder caused by expression of a mutant variant of ataxin-3 (ATX3) protein. Inhibiting expression of ATX3 would provide a therapeutic strategy, but indiscriminant inhibition of both wild-type and mutant ATX3 might lead to undesirable side effects. An ideal silencing agent would block expression of mutant ATX3 while leaving expression of wild-type ATX3 intact. We have previously observed that pepti ...[more]