Article
Allele-specific knockdown of ALS-associated mutant TDP-43 in neural stem cells derived from induced pluripotent stem cells.
PloS one - 1 Jan 2014
Nishimura Agnes L, Shum Carole, Scotter Emma L, Abdelgany Amr, Sardone Valentina, Wright Jamie, Lee Youn-Bok, Chen Han-Jou, Bilican Bilada, Carrasco Monica, Maniatis Tom, Chandran Siddharthan, Rogelj Boris, Gallo Jean-Marc, Shaw Christopher E
Abstract excerpt
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 M337V, which is one of the most common mutations in TDP-43. In order to investigate the use of allele-specific RNA...
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