Human Induced Pluripotent Stem Cell-Derived TDP-43 Mutant Neurons Exhibit Consistent Functional Phenotypes Across Multiple Gene Edited Lines Despite Transcriptomic and Splicing Discrepancies

Human Induced Pluripotent Stem Cell-Derived TDP-43 Mutant Neurons Exhibit Consistent Functional Phenotypes Across Multiple Gene Edited Lines Despite Transcriptomic and Splicing Discrepancies

Gene editing technologies hold great potential to enhance our ability to model inheritable neurodegenerative diseases. Specifically, engineering multiple amyotrophic lateral sclerosis (ALS) mutations into isogenic cell populations facilitates determination of whether different causal mutations cause...

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Bibliographic Details
Main Authors: Alec S. T. Smith, Changho Chun, Jennifer Hesson, Julie Mathieu, Paul N. Valdmanis, David L. Mack, Byung-Ok Choi, Deok-Ho Kim, Mark Bothwell
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-09-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
ALS (amyotrophic lateral sclerosis)
iPSC (induced pluripotent stem cell)
transcriptomics
electrophysiologic analysis
disease model
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2021.728707/full

Internet

https://www.frontiersin.org/articles/10.3389/fcell.2021.728707/full

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