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

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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Bibliographic Details
Main Authors: Xiaohong Xu, Yilin Tay, Bernice Sim, Su-In Yoon, Yihui Huang, Jolene Ooi, Kagistia Hana Utami, Amin Ziaei, Bryan Ng, Carola Radulescu, Donovan Low, Alvin Yu Jin Ng, Marie Loh, Byrappa Venkatesh, Florent Ginhoux, George J. Augustine, Mahmoud A. Pouladi
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Stem Cell Reports
Subjects:
human induced pluripotent stem cell (hiPSC)
neurodegenerative disorders
Huntington disease
genetic editing
disease modeling
disease phenotypes
transcriptional dysrgulation
CHCHD2
mitochondrial dysfunction
Online Access:http://www.sciencedirect.com/science/article/pii/S2213671117300383

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http://www.sciencedirect.com/science/article/pii/S2213671117300383

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