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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Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha: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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