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