Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent-Reference-Cited by-同舟云学术

Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent

Published:2017-10-24 Issue:23 Volume:1 Page:2088-2104
ISSN:2473-9529
Container-title:Blood Advances
language:en
Short-container-title:

Author:

Lévy Camille¹²,Amirache Fouzia¹²,Girard-Gagnepain Anais¹²,Frecha Cecilia³,Roman-Rodríguez Francisco J.⁴⁵⁶,Bernadin Ornellie¹²,Costa Caroline¹²,Nègre Didier¹²,Gutierrez-Guerrero Alejandra¹²,Vranckx Lenard S.⁷,Clerc Isabelle⁸⁹,Taylor Naomi⁸⁹,Thielecke Lars¹⁰,Cornils Kerstin¹¹,Bueren Juan A.⁴⁵⁶,Rio Paula⁴⁵⁶,Gijsbers Rik⁷,Cosset François-Loïc¹²,Verhoeyen Els¹²¹²

Affiliation:

1. International Center for Infectiology Research, Team Enveloped Viruses, Vectors and Innate Responses, INSERM, U1111, Université Claude Bernard Lyon 1, Centre National de la Recherche Scientifique, Unité Mixte de Recherche (UMR) 5308, Ecole Normale Supérieure de Lyon, Lyon, France;

2. LabEx Ecofect, Université de Lyon, Lyon, France;

3. Laboratory of Viral Vectors and Gene Therapy, Institute of Basic Sciences and Experimental Medicine, Italian Hospital School of Medicine Argentina, Buenos Aires, Argentina;

4. Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain;

5. Advanced Therapies Unit, Instituto de Investigación Sanitaria Fundación Jiménez Díaz, Madrid, Spain;

6. Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain;

7. Laboratory for Viral Vector Technology & Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, Katholieke Universiteit Leuven, Leuven, Belgium;

8. Institut de Génétique Moléculaire de Montpellier, Centre National de la Recherche Scientifique UMR5535, Universités de Montpellier 1 et 2, Montpellier, France;

9. Laboratory of Excellence GR-Ex, Paris, France;

10. Institute for Medical Informatics and Biometry, Faculty of Medicine Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany;

11. Department of Pediatric Hematology and Oncology, Division of Pediatric Stem Cell Transplantation and Immunology, Research Institute Children’s Cancer Center Hamburg, Hamburg, Germany; and

12. INSERM, U1065, Centre Méditerranéen de Médecine Moléculaire, Équipe “Contrôle Métabolique des Morts Cellulaires,” Nice, France

Abstract

Key PointsH/F-LVs permit very-high-level transduction of human HSCs, even in the complete absence of cytokine stimulation. H/F-LVs outperform VSV-G-LVs for efficient correction of Fanconi anemia patient cells in unfractionated BM.

Publisher

American Society of Hematology

Subject

Hematology

Link

http://ashpublications.org/bloodadvances/article-pdf/1/23/2088/879000/advances007773.pdf

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