ArticleScience translational medicine2024
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
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Article in Science translational medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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0cells of the map it votes in
14citing papers in PubMed
4.7field-weighted citation impact, top 4% of its field
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Who cites it
14 citing papers in PubMed, 20 citations in OpenAlex.
- RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids.Blood advances · 2025Trial
- In-depth characterization of stem cell potency and genotoxicity for clinical-scaleMolecular therapy. Advances · 2026Article
- ABO: A 3D stroma-supported culture platform enabling full human B-lymphopoiesis for disease modeling and gene therapy development.Cell reports. Medicine · 2026Article
- Modeling and correction of SCID-X1 using CRISPR-Cas9 homology-directed repair in human HSPCs.Molecular therapy. Nucleic acids · 2026Article
- Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters.Nature biotechnology · 2026Article
- Beyond survival: Multisystem long-term outcomes following HSCT in chronic granulomatous disease.Journal of human immunity · 2026Article
- CRISPR-based therapeutic genome editing for inherited blood disorders.Nature reviews. Drug discovery · 2025Review
- Double-hit RAG2 mutation presenting with hyper-immunoglobulin E and preserved T cells diagnostic challenge: a case report.Journal of medical case reports · 2025Article
- Gene therapy for inborn errors of immunity: past progress, current status and future directions.Rare disease and orphan drugs journal · 2025Article
- Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- A p38 MAPK-ROS axis fuels proliferation stress and DNA damage during CRISPR-Cas9 gene editing in hematopoietic stem and progenitor cells.Cell reports. Medicine · 2024Article
- Rediscovering the human thymus through cutting-edge technologies.The Journal of experimental medicine · 2024Review
- TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Review
Corrections and comments
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Authors and funding
34 authors at 7 institutions in 2 countries.
Maria Carmina CastielloSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0003-2581-9648
Chiara BrandasSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0003-1548-6720
Samuele FerrariSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0003-1122-1231
Simona PorcelliniSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0009-0002-6179-6287
Nicolò SacchettiSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.
Daniele CanaruttoSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0001-6999-8479
Elena DraghiciSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.
Ivan MerelliSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0003-3587-3680
Matteo BarcellaSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.
Gabriele PelosiSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.
Valentina VavassoriSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.
Angelica VaresiSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.
Aurelien JacobSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0002-4799-3711
Serena ScalaSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0002-8579-3809
Luca Basso RicciSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0002-5907-005X
Marianna PaulisMilan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.ORCID 0000-0001-7803-9982
Dario StrinaMilan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.
Martina Di VerniereSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0009-0000-0352-1993
Lucia Sergi SergiSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0002-8631-7433
Marta SerafiniTranslational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy.
Steven M HollandLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.ORCID 0000-0003-3207-5464
Jenna R E BergersonLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.
Suk See De RavinLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-9800-774X
Harry L MalechLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.ORCID 0000-0001-5874-5775
Francesca PalaLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.ORCID 0000-0003-4537-9061
Marita BosticardoLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-1771-4523
Chiara BrombinUniversity Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy.ORCID 0000-0002-8215-6206
Federica CugnataUniversity Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy.ORCID 0000-0002-1886-569X
Enrica CalzoniSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0003-4080-2350
Gay M CrooksDepartment of Pathology and Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles (UCLA), Los Angeles, CA 90095, USA.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-8335-0262
Pietro GenoveseSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0001-9507-9003
Luigi NaldiniSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0002-7835-527X
Anna VillaSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.ORCID 0000-0003-4428-9013
The San Raffaele Telethon Institute for Gene Therapy · ITNational Institutes of Health · USVita-Salute San Raffaele University · ITInstitute of Genetic and Biomedical Research · ITHumanitas University · ITAzienda Ospedaliera San Gerardo · ITUniversity of California, Los Angeles · US
Funding
Transgenic Animal Models Of Human Immune DefectsZIAAI000646 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI HOLLAND, STEVEN · 2009 to 2025
$22.2MCharacterization of the cellular and molecular bases of inborn errors of immunityZIAAI001222 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI NOTARANGELO, LUIGI · 2017 to 2025
$15.2MGene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1MDevelopment and Clinical Application of Gene and Cell therapies for Patients with Immune DeficienciesZIAAI000645 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK SEE · 2009 to 2025
$10.1MDevelopment, Optimization and Preclinical Modeling of Hematopoietic Stem Cell Gene Editing for the Treatment of RAG1 ImmunodeficiencyR01AI155796 · NIAID · DANA-FARBER CANCER INST · PI GENOVESE, PIETRO · 2021 to 2025
$2.2MIntramural NIH HHS ZIA AI001222NIAID NIH HHS R01 AI155796
Abstract
Recombination activating genes (
Indexed as
Gene EditingHematopoietic Stem Cell TransplantationAnimalsExonsHematopoietic Stem CellsHomeodomain ProteinsHumansMiceHomeodomain ProteinsRAG-1 protein
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