Identification of Compounds that Rescue IKBKAP Expression in Familial Dysautonomia-iPS Cells

Patient-specific induced pluripotent stem cells (iPSCs) represent a novel system for modeling human genetic disease and could develop into a key drug discovery platform. We recently reported disease-specific phenotypes in iPSCs from familial dysautonomia (FD) patients. FD is a rare but fatal genetic...

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Detalles Bibliográficos
Autores principales: Lee, Gabsang, Ramirez, Christina N., Kim, Hyesoo, Zeltner, Nadja, Liu, Becky, Radu, Constantin, Bhinder, Bhavneet, Kim, Yong Jun, Choi, InYoung, Mukherjee-Clavin, Bipasha, Djaballah, Hakim, Studer, Lorenz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2012
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3711177/
https://www.ncbi.nlm.nih.gov/pubmed/23159879
http://dx.doi.org/10.1038/nbt.2435

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3711177/
https://www.ncbi.nlm.nih.gov/pubmed/23159879
http://dx.doi.org/10.1038/nbt.2435

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