Article
Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs.
Nature - 17 Sept 2009
Lee Gabsang, Papapetrou Eirini P, Kim Hyesoo, Chambers Stuart M, Tomishima Mark J, Fasano Christopher A, Ganat Yosif M, Menon Jayanthi, Shimizu Fumiko, Viale Agnes, Tabar Viviane, Sadelain Michel, Studer Lorenz
Abstract excerpt
The isolation of human induced pluripotent stem cells (iPSCs) offers a new strategy for modelling human disease. Recent studies have reported the derivation and differentiation of disease-specific human iPSCs. However, a key challenge in the field is the demonstration of disease-related phenotypes and the ability to model pathogenesis and treatment of disease in iPSCs. Familial dysautonomia (FD) is a rare but...
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