Ontology highlight
ABSTRACT:
SUBMITTER: Fink KD
PROVIDER: S-EPMC6476541 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
Fink Kyle D KD Deng Peter P Gutierrez Josh J Anderson Joseph S JS Torrest Audrey A Komarla Anvita A Kalomoiris Stefanos S Cary Whitney W Anderson Johnathon D JD Gruenloh William W Duffy Alexandra A Tempkin Teresa T Annett Geralyn G Wheelock Vicki V Segal David J DJ Nolta Jan A JA
Cell transplantation 20160204 4
Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder caused by an abnormal expansion of CAG repeats. Although pathogenesis has been attributed to this polyglutamine expansion, the underlying mechanisms through which the huntingtin protein functions have yet to be elucidated. It has been suggested that postnatal reduction of mutant huntingtin through protein interference or conditional gene knockout could prove to be an effective therapy for patients suffering from HD. Fo ...[more]