Article
Human amyotrophic lateral sclerosis excitability phenotype screen: Target discovery and validation.
Cell reports - 8 Jun 2021
Huang Xuan, Roet Kasper C D, Zhang Liying, Brault Amy, Berg Allison P, Jefferson Anne B, Klug-McLeod Jackie, Leach Karen L, Vincent Fabien, Yang Hongying, Coyle Anthony J, Jones Lyn H, Frost Devlin, Wiskow Ole, Chen Kuchuan, Maeda Rie, Grantham Alyssa, Dornon Mary K, Klim Joseph R, Siekmann Marco T, Zhao Dongyi, Lee Seungkyu, Eggan Kevin, Woolf Clifford J
Abstract excerpt
Drug development is hampered by poor target selection. Phenotypic screens using neurons differentiated from patient stem cells offer the possibility to validate known and discover novel disease targets in an unbiased fashion. To identify targets for managing hyperexcitability, a pathological feature of amyotrophic lateral sclerosis (ALS), we design a multi-step screening funnel using patient-derived motor...
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