@article{54e4c1de4f4a4f3a8dec26b9735b3af5,
title = "Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A",
abstract = "Follenzi and colleagues show that endothelial cells can be obtained starting from blood CD34+ cells of both healthy and hemophilic donors passing through iPSC generation. During the differentiation process the cells can be genetically modified by LV to express FVIII; corrected endothelial cells secrete FVIII in vitro and ameliorate the hemophilic phenotype in a mouse model of the disease.",
keywords = "FVIII, cell and gene therapy, chimeric vasculature, endothelial cells, hemophilia A, induced pluripotent stem cells (iPSCs), lentiviral vectors",
author = "Cristina Olgasi and Maria Talmon and Simone Merlin and Alessia Cucci and Yvonne Richaud-Patin and Gabriella Ranaldo and Donato Colangelo and \{Di Scipio\}, Federica and Berta, \{Giovanni N.\} and Chiara Borsotti and Federica Valeri and Francesco Faraldi and Maria Prat and Maria Messina and Piercarla Schinco and Angelo Lombardo and Angel Raya and Antonia Follenzi",
note = "Publisher Copyright: {\textcopyright} 2018 The Authors",
year = "2018",
month = dec,
day = "11",
doi = "10.1016/j.stemcr.2018.10.012",
language = "English",
volume = "11",
pages = "1391--1406",
journal = "Stem Cell Reports",
issn = "2213-6711",
publisher = "Cell Press",
number = "6",
}