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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...
Autores principales: | , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2011
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3134447/ https://www.ncbi.nlm.nih.gov/pubmed/21765802 http://dx.doi.org/10.1371/journal.pbio.1001099 |