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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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| Hoofdauteurs: | , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
American Society of Hematology
2011
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| Onderwerpen: | |
| Online toegang: | 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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