Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts

Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts

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 elucid...

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Bibliographic Details
Main Authors: Kyle D. Fink Ph.D, Peter Deng, Josh Gutierrez, Joseph S. Anderson, Audrey Torrest, Anvita Komarla, Stefanos Kalomoiris, Whitney Cary, Johnathon D. Anderson, William Gruenloh, Alexandra Duffy, Teresa Tempkin, Geralyn Annett, Vicki Wheelock, David J. Segal, Jan A. Nolta
Format: Article
Language:English
Published: SAGE Publishing 2016-04-01
Series:Cell Transplantation
Online Access:https://doi.org/10.3727/096368916X690863

Internet

https://doi.org/10.3727/096368916X690863

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