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Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.

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Title: Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.
Authors: Rigamonti C; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Vita-Salute San Raffaele University, 20132 Milan, Italy., Bulté D; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Barzaghi F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Mesa-Nuñez C; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Basso-Ricci L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Pettinato E; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Visconti C; Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Degl'Innocenti S; GLP Test Facility, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Gazzo F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Department of Electronics, Information and Bioengineering, Politecnico di Milano, 20133 Milan, Italy., Benedicenti F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Arévalo S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Quaranta P; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Sanvito F; GLP Test Facility, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Pathology Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Caorsi R; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy., Schena F; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy., Lupia M; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy., Federici S; Division of Rheumatology, Bambino Gesù Children's Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy., Insalaco A; Division of Rheumatology, Bambino Gesù Children's Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy., De Benedetti F; Division of Rheumatology, Bambino Gesù Children's Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy., Scala S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Miano M; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy., Conti F; Pediatric Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, 40138 Bologna, Italy.; Department of Medical and Surgical Sciences, Alma Mater Studiorum, University of Bologna, 40126 Bologna, Italy., Cicalese MP; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Montini E; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Gattorno M; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy., Dufour C; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy., Aiuti A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Mortellaro A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Oct 30; Vol. 33 (4), pp. 101624. Date of Electronic Publication: 2025 Oct 30 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2329-0501
DOI:10.1016/j.omtm.2025.101624