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 |
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