Unbiased profiling of isogenic huntington disease hPSC-derived CNS and peripheral cells reveals strong cell-type specificity of CAG length effects

• Main Authors: Ziaei, Amin, Zeng, Ruizhu, Low, Donovan, Aminkeng, Folefac, Sobota, Radoslaw M., Ginhoux, Florent, Petretto, Enrico, Pouladi, Mahmoud A., Langley, Sarah Raye, Ooi, Jolene, Xu, Xiaohong, Utami, Kagistia H., Sim, Bernice, Huang, Yihui, Harmston, Nathan P., Tay, Yi Lin
• Other Authors: Lee Kong Chian School of Medicine (LKCMedicine)
• Format: Article
• Language: English
• Published: 2019
• Subjects: CAG Repeat; DRNTU::Science::Medicine; Huntington Disease
• Online Access: https://hdl.handle.net/10356/93136; http://hdl.handle.net/10220/48521
• Institution: Nanyang Technological University

In Huntington disease (HD), the analysis of tissue-specific CAG repeat length effects has been challenging, given the difficulty in obtaining relevant patient tissues with a broad range of CAG repeat lengths. We used genome editing to generate an allelic panel of isogenic HD (IsoHD) human embryonic...