Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A

We generated patient-specific disease-free induced pluripotent stem cells (iPSCs) from peripheral blood CD34+ cells and differentiated them into functional endothelial cells (ECs) secreting factor VIII (FVIII) for gene and cell therapy approaches to cure hemophilia A (HA), an X-linked bleeding disor...

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Detalhes bibliográficos
Publicado no:Stem Cell Reports
Main Authors: Olgasi, Cristina, Talmon, Maria, Merlin, Simone, Cucci, Alessia, Richaud-Patin, Yvonne, Ranaldo, Gabriella, Colangelo, Donato, Di Scipio, Federica, Berta, Giovanni N., Borsotti, Chiara, Valeri, Federica, Faraldi, Francesco, Prat, Maria, Messina, Maria, Schinco, Piercarla, Lombardo, Angelo, Raya, Angel, Follenzi, Antonia
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6294075/
https://ncbi.nlm.nih.gov/pubmed/30416049
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2018.10.012
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