Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome-Reference-Cited by-同舟云学术

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome

Published:2021-01-21 Issue:3 Volume:13 Page:
ISSN:1757-4676
Container-title:EMBO Molecular Medicine
language:en
Short-container-title:EMBO Mol Med

Author:

Vavassori Valentina¹²,Mercuri Elisabetta¹³,Marcovecchio Genni E¹,Castiello Maria C¹⁴,Schiroli Giulia¹,Albano Luisa¹,Margulies Carrie⁵,Buquicchio Frank⁵,Fontana Elena⁴⁶,Beretta Stefano¹,Merelli Ivan¹⁷,Cappelleri Andrea⁸⁹,Rancoita Paola MV¹⁰,Lougaris Vassilios¹¹,Plebani Alessandro¹¹,Kanariou Maria¹²,Lankester Arjan¹³,Ferrua Francesca¹¹⁴,Scanziani Eugenio⁸⁹,Cotta‐Ramusino Cecilia⁵,Villa Anna¹⁴,Naldini Luigi¹²^ORCID,Genovese Pietro¹^ORCID

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy IRCCS San Raffaele Scientific Institute Milan Italy

2. Vita‐Salute San Raffaele University Milan Italy

3. Milano‐Bicocca University Monza Italy

4. Institute of Genetic and Biomedical Research Milan Unit National Research Council (CNR) Milan Italy

5. Editas Medicine Cambridge MA USA

6. Human Genome Lab Humanitas Clinical and Research Center Milan Italy

7. Institute for Biomedical Technologies National Research Council (CNR) Segrate Italy

8. Mouse and Animal Pathology Laboratory (MAPLab) Fondazione Unimi Milano Italy

9. Department of Veterinary Medicine University of Milan Milan Italy

10. University Center for Statistics in the Biomedical Sciences (CUSSB) Vita‐Salute San Raffaele University Milan Italy

11. University of Brescia and ASST‐Spedali Civili di Brescia Brescia Italy

12. First Department of Paediatrics Aghia Sophia Children's Hospital Athens Greece

13. Department of Pediatrics Leiden University Medical Center Leiden The Netherlands

14. Pediatric Immunohematology and Bone Marrow Transplantation Unit IRCCS San Raffaele Scientific Institute Milan Italy

Funder

Fondazione Telethon

Ministero della Salute

Banca d'Italia

Ministero dell’Istruzione, dell’Università e della Ricerca

Louis-Jeantet Foundation

Publisher

EMBO

Subject

Molecular Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.202013545

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