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Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease
Human induced pluripotent stem cells (iPSCs) bearing monogenic mutations have great potential for modeling disease phenotypes, screening candidate drugs, and cell replacement therapy provided the underlying disease-causing mutation can be corrected. Here, we report a homologous recombination-based a...
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| Autors principals: | , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society of Hematology
2011
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3208277/ https://ncbi.nlm.nih.gov/pubmed/21881051 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2011-02-335554 |
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