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Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression.

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Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression.
Lee G, Ramirez CN, Kim H, Zeltner N, Liu B, Radu C, Bhinder B, Kim Y J, Choi I Y, Mukherjee-Clavin B, Djaballah H, Studer L
Nat Biotechnol. 2012 Dec; 30(12):1244-8. Epub 2012 Nov 25.
PMID: 23159879.
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