Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells

Huntington disease (HD) is a dominant neurodegenerative disorder caused by a CAG repeat expansion in HTT. Here we report correction of HD human induced pluripotent stem cells (hiPSCs) using a CRISPR-Cas9 and piggyBac transposon-based approach. We show that both HD and corrected isogenic hiPSCs can b...

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Detalles Bibliográficos
Autores principales: Xu, Xiaohong, Tay, Yilin, Sim, Bernice, Yoon, Su-In, Huang, Yihui, Ooi, Jolene, Utami, Kagistia Hana, Ziaei, Amin, Ng, Bryan, Radulescu, Carola, Low, Donovan, Ng, Alvin Yu Jin, Loh, Marie, Venkatesh, Byrappa, Ginhoux, Florent, Augustine, George J., Pouladi, Mahmoud A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5355646/
https://www.ncbi.nlm.nih.gov/pubmed/28238795
http://dx.doi.org/10.1016/j.stemcr.2017.01.022

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