Article
Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells.
Stem cell reports - 14 Mar 2017
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
Abstract excerpt
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 be differentiated into excitable, synaptically active forebrain neurons. We further demonstrate that phenotypic...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.