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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 elucidated....

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Bibliografske podrobnosti
izdano v:Cell Transplant
Main Authors: 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.
Format: Artigo
Jezik:Inglês
Izdano: 2016
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6476541/
https://ncbi.nlm.nih.gov/pubmed/26850319
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3727/096368916X690863
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