Ontology highlight
ABSTRACT:
SUBMITTER: Ramachandran PS
PROVIDER: S-EPMC3701762 | biostudies-literature | 2013 Jul
REPOSITORIES: biostudies-literature
Ramachandran Pavitra S PS Keiser Megan S MS Davidson Beverly L BL
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 20130701 3
Over the last decade, RNA interference technology has shown therapeutic promise in rodent models of dominantly inherited brain diseases, including those caused by polyglutamine repeat expansions in the coding region of the affected gene. For some of these diseases, proof-of concept studies in model organisms have transitioned to safety testing in larger animal models, such as the nonhuman primate. Here, we review recent progress on RNA interference-based therapies in various model systems. We al ...[more]