View in EDS HTML Full Text PDF Full Text

Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.

Saved in:
Bibliographic Details
Title: Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Authors: Castiello MC; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy., Brandas C; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Translational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy., Ferrari S; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Porcellini S; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Sacchetti N; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy., Canarutto D; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy.; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy., Draghici E; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Merelli I; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; National Research Council (CNR), Institute for Biomedical Technologies, Segrate (MI) 20054, Italy., Barcella M; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; National Research Council (CNR), Institute for Biomedical Technologies, Segrate (MI) 20054, Italy., Pelosi G; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy., Vavassori V; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Varesi A; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Jacob A; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Scala S; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Basso Ricci L; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Paulis M; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.; Humanitas Clinical and Research Center IRCCS, Rozzano (MI) 20089, Italy., Strina D; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.; Humanitas Clinical and Research Center IRCCS, Rozzano (MI) 20089, Italy., Di Verniere M; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy., Sergi Sergi L; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Serafini M; Translational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy.; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza (MI) 20900, Italy., Holland SM; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Bergerson JRE; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., De Ravin SS; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Malech HL; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Pala F; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Bosticardo M; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Brombin C; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy., Cugnata F; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy., Calzoni E; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Crooks GM; Department of Pathology and Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles (UCLA), Los Angeles, CA 90095, USA., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Genovese P; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA 02115, USA., Naldini L; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy., Villa A; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.
Source: Science translational medicine [Sci Transl Med] 2024 Feb 07; Vol. 16 (733), pp. eadh8162. Date of Electronic Publication: 2024 Feb 07.
Publication Type: Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Full text is not displayed to guests.
Description
ISSN:1946-6242
DOI:10.1126/scitranslmed.adh8162