Allele-specific knockdown of ALS-associated mutant TDP-43 in neural stem cells derived from induced pluripotent stem cells.

Allele-specific knockdown of ALS-associated mutant TDP-43 in neural stem cells derived from induced pluripotent stem cells.

TDP-43 is found in cytoplasmic inclusions in 95% of amyotrophic lateral sclerosis (ALS) and 60% of frontotemporal lobar degeneration (FTLD). Approximately 4% of familial ALS is caused by mutations in TDP-43. The majority of these mutations are found in the glycine-rich domain, including the variant...

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Main Authors: Agnes L Nishimura, Carole Shum, Emma L Scotter, Amr Abdelgany, Valentina Sardone, Jamie Wright, Youn-Bok Lee, Han-Jou Chen, Bilada Bilican, Monica Carrasco, Tom Maniatis, Siddharthan Chandran, Boris Rogelj, Jean-Marc Gallo, Christopher E Shaw
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2014-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0091269
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https://doi.org/10.1371/journal.pone.0091269

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