Ontology highlight
ABSTRACT:
SUBMITTER: Munoz-Sanjuan I
PROVIDER: S-EPMC3026740 | biostudies-other | 2011 Feb
REPOSITORIES: biostudies-other
The Journal of clinical investigation 20110201 2
Huntington disease (HD) is a dominantly inherited neurodegenerative disorder that results from expansion of the polyglutamine repeat in the huntingtin (HTT) gene. There are currently no effective treatments for this devastating disease. Given its monogenic nature, disease modification therapies for HD should be theoretically feasible. Currently, pharmacological therapies aimed at disease modification by altering levels of HTT protein are in late-stage preclinical development. Here, we review cur ...[more]