Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice.
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| Title: | Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice. |
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| Authors: | Hong Y; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK. y.hong@ucl.ac.uk., Burleigh A; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Liao A; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Yeung J; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Bian Y; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.; Department of Clinical Laboratory, Qilu Hospital of Shandong University Dezhou Hospital, Dezhou, China., Sebire N; Department of Histopathology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Ogunbiyi O; Department of Histopathology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Omoyinmi E; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Thrasher AJ; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Morris E; UCL Institute of Immunity & Transplantation, London, UK., Brogan PA; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Eleftheriou D; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK. |
| Source: | Gene therapy [Gene Ther] 2025 Dec; Vol. 32 (6), pp. 664-671. Date of Electronic Publication: 2025 Jun 24. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 40555830 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Hong+Y%22">Hong Y</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK. y.hong@ucl.ac.uk.<br /><searchLink fieldCode="AU" term="%22Burleigh+A%22">Burleigh A</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Liao+A%22">Liao A</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Yeung+J%22">Yeung J</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Bian+Y%22">Bian Y</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.; Department of Clinical Laboratory, Qilu Hospital of Shandong University Dezhou Hospital, Dezhou, China.<br /><searchLink fieldCode="AU" term="%22Sebire+N%22">Sebire N</searchLink>; Department of Histopathology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Ogunbiyi+O%22">Ogunbiyi O</searchLink>; Department of Histopathology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Omoyinmi+E%22">Omoyinmi E</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Thrasher+AJ%22">Thrasher AJ</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Morris+E%22">Morris E</searchLink>; UCL Institute of Immunity & Transplantation, London, UK.<br /><searchLink fieldCode="AU" term="%22Brogan+PA%22">Brogan PA</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Eleftheriou+D%22">Eleftheriou D</searchLink>; Infection, Immunity, Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%229421525%22">Gene therapy</searchLink> [Gene Ther] 2025 Dec; Vol. 32 (6), pp. 664-671. <i>Date of Electronic Publication: </i>2025 Jun 24. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article; Research Support, Non-U.S. Gov't – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Group%22">Nature Publishing Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9421525 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1476-5462 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209697128%22">09697128 </searchLink><i>NLM ISO Abbreviation: </i>Gene Ther <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=40555830 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1038/s41434-025-00547-4 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 664 Titles: – TitleFull: Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Hong Y – PersonEntity: Name: NameFull: Burleigh A – PersonEntity: Name: NameFull: Liao A – PersonEntity: Name: NameFull: Yeung J – PersonEntity: Name: NameFull: Bian Y – PersonEntity: Name: NameFull: Sebire N – PersonEntity: Name: NameFull: Ogunbiyi O – PersonEntity: Name: NameFull: Omoyinmi E – PersonEntity: Name: NameFull: Thrasher AJ – PersonEntity: Name: NameFull: Morris E – PersonEntity: Name: NameFull: Brogan PA – PersonEntity: Name: NameFull: Eleftheriou D IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 12 Text: 2025 Dec Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 1476-5462 Numbering: – Type: volume Value: 32 – Type: issue Value: 6 Titles: – TitleFull: Gene therapy Type: main |
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