Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections

Author:

Hetzel Miriam12,Mucci Adele12,Blank Patrick3,Nguyen Ariane Hai Ha14,Schiller Jan14,Halle Olga5,Kühnel Mark-Philipp6,Billig Sandra7,Meineke Robert14,Brand Daniel1,Herder Vanessa8,Baumgärtner Wolfgang8,Bange Franz-Christoph7,Goethe Ralph9,Jonigk Danny6,Förster Reinhold5,Gentner Bernhard10,Casanova Jean-Laurent1112131415,Bustamante Jacinta11121316,Schambach Axel117,Kalinke Ulrich3,Lachmann Nico14ORCID

Affiliation:

1. Institute of Experimental Hematology and

2. Research Group Reprogramming and Gene Therapy, REBIRTH Cluster-of Excellence, Hannover Medical School, Hannover, Germany;

3. Institute for Experimental Infection Research, TWINCORE, Centre for Experimental and Clinical Infection Research, Hannover, Germany;

4. Young Research Group Translational Hematology of Congenital Diseases, REBIRTH Cluster-of Excellence,

5. Institute of Immunology,

6. Institute for Pathology, and

7. Institute of Medical Microbiology and Hospital Epidemiology, Hannover Medical School, Hannover, Germany;

8. Department of Pathology and

9. Institute for Microbiology, Department of Infectious Diseases, University of Veterinary Medicine Hannover, Hannover, Germany;

10. San Raffaele Telethon Institute for Gene Therapy, Scientific Institute Hospital San Raffaele, Milan, Italy;

11. Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Hospital for Sick Children, Paris, France;

12. Paris Descartes University, Imagine Institute, Paris, France;

13. St. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, NY;

14. Howard Hughes Medical Institute, New York, NY;

15. Pediatric Hematology-Immunology Unit and

16. Center for the Study of Primary Immunodeficiencies, Assistance Publique–Hôpitaux de Paris, Necker Hospital for Sick Children, Paris, France; and

17. Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA

Abstract

Key Points Hematopoietic cell and gene therapy can prevent in vivo infections by Mycobacteria spp. using different lentiviral vectors. Cellular repair of macrophages in vivo highlights phagocytes as key players in the disease progression.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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