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 en:Cell Transplant
Autores principales: 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
Lenguaje:Inglês
Publicado: 2016
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Article
Acceso en línea: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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