Article
Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts.
Cell transplantation - 1 Jan 2016
Fink Kyle D, Deng Peter, Gutierrez Josh, Anderson Joseph S, Torrest Audrey, Komarla Anvita, Kalomoiris Stefanos, Cary Whitney, Anderson Johnathon D, Gruenloh William, Duffy Alexandra, Tempkin Teresa, Annett Geralyn, Wheelock Vicki, Segal David J, Nolta Jan A
Abstract excerpt
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...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.