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

• Main Authors: Augustine, George James, Pouladi, Mahmoud A., 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
• Other Authors: Lee Kong Chian School of Medicine (LKCMedicine)
• Format: Article
• Language: English
• Published: 2017
• Subjects: CHCHD2; hiPSC
• Online Access: https://hdl.handle.net/10356/86517; http://hdl.handle.net/10220/44036
• Institution: Nanyang Technological University

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...