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

Cristina Olgasi; Maria Talmon; Simone Merlin; Alessia Cucci; Yvonne Richaud-Patin; Gabriella Ranaldo; Donato Colangelo; Federica Di Scipio; Giovanni N. Berta; Chiara Borsotti; Federica Valeri; Francesco Faraldi; Maria Prat; Maria Messina; Piercarla Schinco; Angelo Lombardo; Angel Raya; Antonia Follenzi

doi:10.1016/j.stemcr.2018.10.012

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

Cristina Olgasi, Maria Talmon, Simone Merlin, Alessia Cucci, Yvonne Richaud-Patin, Gabriella Ranaldo, Donato Colangelo, Federica Di Scipio, Giovanni N. Berta, Chiara Borsotti, Federica Valeri, Francesco Faraldi, Maria Prat, Maria Messina, Piercarla Schinco, Angelo Lombardo, Angel Raya, Antonia Follenzi

Research output: Contribution to journal › Article › peer-review

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.

Original language	English
Pages (from-to)	1391-1406
Number of pages	16
Journal	Stem Cell Reports
Volume	11
Issue number	6
DOIs	https://doi.org/10.1016/j.stemcr.2018.10.012
Publication status	Published - 11 Dec 2018

Keywords

FVIII
cell and gene therapy
chimeric vasculature
endothelial cells
hemophilia A
induced pluripotent stem cells (iPSCs)
lentiviral vectors

Access to Document

10.1016/j.stemcr.2018.10.012

Cite this

Olgasi, C., Talmon, M., Merlin, S., Cucci, A., Richaud-Patin, Y., Ranaldo, G., Colangelo, D., Di Scipio, F., Berta, G. N., Borsotti, C., Valeri, F., Faraldi, F., Prat, M., Messina, M., Schinco, P., Lombardo, A., Raya, A., & Follenzi, A. (2018). Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A. Stem Cell Reports, 11(6), 1391-1406. https://doi.org/10.1016/j.stemcr.2018.10.012

@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",

}

Olgasi, C , Talmon, M , Merlin, S, Cucci, A, Richaud-Patin, Y, Ranaldo, G, Colangelo, D, Di Scipio, F, Berta, GN, Borsotti, C, Valeri, F, Faraldi, F, Prat, M, Messina, M, Schinco, P, Lombardo, A, Raya, A & Follenzi, A 2018, 'Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A', Stem Cell Reports, vol. 11, no. 6, pp. 1391-1406. https://doi.org/10.1016/j.stemcr.2018.10.012

TY - JOUR

T1 - Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A

AU - Olgasi, Cristina

AU - Talmon, Maria

AU - Merlin, Simone

AU - Cucci, Alessia

AU - Richaud-Patin, Yvonne

AU - Ranaldo, Gabriella

AU - Colangelo, Donato

AU - Di Scipio, Federica

AU - Berta, Giovanni N.

AU - Borsotti, Chiara

AU - Valeri, Federica

AU - Faraldi, Francesco

AU - Prat, Maria

AU - Messina, Maria

AU - Schinco, Piercarla

AU - Lombardo, Angelo

AU - Raya, Angel

AU - Follenzi, Antonia

PY - 2018/12/11

Y1 - 2018/12/11

N2 - 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.

AB - 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.

KW - FVIII

KW - cell and gene therapy

KW - chimeric vasculature

KW - endothelial cells

KW - hemophilia A

KW - induced pluripotent stem cells (iPSCs)

KW - lentiviral vectors

UR - http://www.scopus.com/inward/record.url?scp=85058609136&partnerID=8YFLogxK

U2 - 10.1016/j.stemcr.2018.10.012

DO - 10.1016/j.stemcr.2018.10.012

M3 - Article

SN - 2213-6711

VL - 11

SP - 1391

EP - 1406

JO - Stem Cell Reports

JF - Stem Cell Reports

IS - 6

ER -

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

Abstract

Keywords

Access to Document

Other files and links

Fingerprint

Cite this