Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs.

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

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.

Affiliation

Lee G; Developmental Biology Program, Sloan-Kettering Institute, 1275 York Ave, USA.

Nature ; 461(7262): 402-6, 2009 Sep 17.

Article in En | MEDLINE | ID: mdl-19693009

Subject(s)

Dysautonomia, Familial/pathology; Dysautonomia, Familial/therapy; Models, Biological; Pluripotent Stem Cells/metabolism; Pluripotent Stem Cells/transplantation; Adolescent; Alternative Splicing/drug effects; Alternative Splicing/genetics; Animals; Carrier Proteins/genetics; Cell Dedifferentiation; Cell Differentiation; Cell Lineage; Cell Movement; Cells, Cultured; Child; Dysautonomia, Familial/drug therapy; Dysautonomia, Familial/genetics; Female; Fibroblasts/cytology; Fibroblasts/metabolism; Gene Expression Profiling; Humans; Kinetin/pharmacology; Kinetin/therapeutic use; Male; Mice; Neural Crest/cytology; Neural Crest/drug effects; Organ Specificity; Phenotype; Pluripotent Stem Cells/cytology; Pluripotent Stem Cells/drug effects; Transcriptional Elongation Factors

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Full text: 1 Database: MEDLINE Main subject: Dysautonomia, Familial / Pluripotent Stem Cells / Models, Biological Type of study: Etiology_studies / Prognostic_studies Limits: Adolescent / Animals / Child / Female / Humans / Male Language: En Journal: Nature Year: 2009 Type: Article Affiliation country: United States

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