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Targeting N-Terminal Huntingtin with a Dual-sgRNA Strategy by CRISPR/Cas9

Huntington's disease (HD) is an autosomal dominant progressive neurodegenerative disorder, caused by a CAG/polyglutamine (polyQ) repeat expansion in the Huntingtin (HTT) gene. The polyQ tract is located in and transcribed from N-terminal HTT of exon 1. HTT is a large multifaceted protein, which...

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Detalles Bibliográficos
Autores principales: Wu, Junjiao, Tang, Yu, Zhang, Chun-Li
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Hindawi 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6881766/
https://www.ncbi.nlm.nih.gov/pubmed/31828084
http://dx.doi.org/10.1155/2019/1039623

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