320 Genetic Compensation as a mechanism underlying patients with Rare ALS

OBJECTIVES/GOALS: Rare mutations in CHCHD10 gene are found in 1% of patients with familial Amyotrophic lateral sclerosis (ALS). The overall goal of this study is to utilize induced pluripotent stem cells (iPSCs) as an in vitro model organism for rare ALS variants to evaluate the mechanism of transcr...

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Bibliographic Details
Main Authors: Adriana Morales Gomez, Nathan Staff, Stephen C. Ekker
Format: Article
Language:English
Published: Cambridge University Press 2022-04-01
Series:Journal of Clinical and Translational Science
Online Access:https://www.cambridge.org/core/product/identifier/S2059866122001789/type/journal_article