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Article

Genetic correction of tauopathy phenotypes in neurons derived from human induced pluripotent stem cells.

Stem cell reports - 1 Jan 2013

Fong Helen, Wang Chengzhong, Knoferle Johanna, Walker David, Balestra Maureen E, Tong Leslie M, Leung Laura, Ring Karen L, Seeley William W, Karydas Anna, Kshirsagar Mihir A, Boxer Adam L, Kosik Kenneth S, Miller Bruce L, Huang Yadong

Abstract excerpt

Tauopathies represent a group of neurodegenerative disorders characterized by the accumulation of pathological TAU protein in brains. We report a human neuronal model of tauopathy derived from induced pluripotent stem cells (iPSCs) carrying a TAU-A152T mutation. Using zinc-finger nuclease-mediated gene editing, we generated two isogenic iPSC lines: one with the mutation corrected, and another with the homozygous...

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Identifiers

PMID: 24319659
DOI: 10.1016/j.stemcr.2013.08.001
PMCID: PMC3849235

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