View in EDS

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

Saved in:
Bibliographic Details
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
FullText Text:
  Availability: 0
Header DbId: mdl
DbLabel: MEDLINE Ultimate
An: 41321395
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AU" term="%22Rigamonti+C%22">Rigamonti C</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Bulté+D%22">Bulté D</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Barzaghi+F%22">Barzaghi F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Mesa-Nuñez+C%22">Mesa-Nuñez C</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Basso-Ricci+L%22">Basso-Ricci L</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Pettinato+E%22">Pettinato E</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Visconti+C%22">Visconti C</searchLink>; Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Degl'Innocenti+S%22">Degl'Innocenti S</searchLink>; GLP Test Facility, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Gazzo+F%22">Gazzo F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Benedicenti+F%22">Benedicenti F</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Arévalo+S%22">Arévalo S</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Quaranta+P%22">Quaranta P</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Sanvito+F%22">Sanvito F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Caorsi+R%22">Caorsi R</searchLink>; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Schena+F%22">Schena F</searchLink>; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Lupia+M%22">Lupia M</searchLink>; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Federici+S%22">Federici S</searchLink>; Division of Rheumatology, Bambino Gesù Children's Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Insalaco+A%22">Insalaco A</searchLink>; Division of Rheumatology, Bambino Gesù Children's Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22De+Benedetti+F%22">De Benedetti F</searchLink>; Division of Rheumatology, Bambino Gesù Children's Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Scala+S%22">Scala S</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Miano+M%22">Miano M</searchLink>; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Conti+F%22">Conti F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Cicalese+MP%22">Cicalese MP</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Montini+E%22">Montini E</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Gattorno+M%22">Gattorno M</searchLink>; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Dufour+C%22">Dufour C</searchLink>; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Aiuti+A%22">Aiuti A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Mortellaro+A%22">Mortellaro A</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22101624857%22">Molecular therapy. Methods & clinical development</searchLink> [Mol Ther Methods Clin Dev] 2025 Oct 30; Vol. 33 (4), pp. 101624. <i>Date of Electronic Publication: </i>2025 Oct 30 (<i>Print Publication: </i>2025).
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101624857 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>2329-0501 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223290501%22">23290501 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther Methods Clin Dev <i>Subsets: </i>PubMed not MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41321395
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1016/j.omtm.2025.101624
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        StartPage: 101624
    Titles:
      – TitleFull: Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Rigamonti C
      – PersonEntity:
          Name:
            NameFull: Bulté D
      – PersonEntity:
          Name:
            NameFull: Barzaghi F
      – PersonEntity:
          Name:
            NameFull: Mesa-Nuñez C
      – PersonEntity:
          Name:
            NameFull: Basso-Ricci L
      – PersonEntity:
          Name:
            NameFull: Pettinato E
      – PersonEntity:
          Name:
            NameFull: Visconti C
      – PersonEntity:
          Name:
            NameFull: Degl'Innocenti S
      – PersonEntity:
          Name:
            NameFull: Gazzo F
      – PersonEntity:
          Name:
            NameFull: Benedicenti F
      – PersonEntity:
          Name:
            NameFull: Arévalo S
      – PersonEntity:
          Name:
            NameFull: Quaranta P
      – PersonEntity:
          Name:
            NameFull: Sanvito F
      – PersonEntity:
          Name:
            NameFull: Caorsi R
      – PersonEntity:
          Name:
            NameFull: Schena F
      – PersonEntity:
          Name:
            NameFull: Lupia M
      – PersonEntity:
          Name:
            NameFull: Federici S
      – PersonEntity:
          Name:
            NameFull: Insalaco A
      – PersonEntity:
          Name:
            NameFull: De Benedetti F
      – PersonEntity:
          Name:
            NameFull: Scala S
      – PersonEntity:
          Name:
            NameFull: Miano M
      – PersonEntity:
          Name:
            NameFull: Conti F
      – PersonEntity:
          Name:
            NameFull: Cicalese MP
      – PersonEntity:
          Name:
            NameFull: Montini E
      – PersonEntity:
          Name:
            NameFull: Gattorno M
      – PersonEntity:
          Name:
            NameFull: Dufour C
      – PersonEntity:
          Name:
            NameFull: Aiuti A
      – PersonEntity:
          Name:
            NameFull: Mortellaro A
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 30
              M: 10
              Text: 2025 Oct 30
              Type: published
              Y: 2025
          Identifiers:
            – Type: issn-print
              Value: 2329-0501
          Numbering:
            – Type: volume
              Value: 33
            – Type: issue
              Value: 4
          Titles:
            – TitleFull: Molecular therapy. Methods & clinical development
              Type: main
ResultId 1