Generation of Healthy Mice from Gene-Corrected Disease-Specific Induced Pluripotent Stem Cells

Using the murine model of tyrosinemia type 1 (fumarylacetoacetate hydrolase [FAH] deficiency; FAH (−/−) mice) as a paradigm for orphan disorders, such as hereditary metabolic liver diseases, we evaluated fibroblast-derived FAH (−/−)-induced pluripotent stem cells (iPS cells) as targets for gene corr...

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Detalhes bibliográficos
Main Authors: Wu, Guangming, Liu, Na, Rittelmeyer, Ina, Sharma, Amar Deep, Sgodda, Malte, Zaehres, Holm, Bleidißel, Martina, Greber, Boris, Gentile, Luca, Han, Dong Wook, Rudolph, Cornelia, Steinemann, Doris, Schambach, Axel, Ott, Michael, Schöler, Hans R., Cantz, Tobias
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2011
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3134447/
https://ncbi.nlm.nih.gov/pubmed/21765802
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pbio.1001099
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