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Safety and efficacy of gene therapy for RAG1-deficient SCID.

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Title: Safety and efficacy of gene therapy for RAG1-deficient SCID.
Authors: Staal FJT; Leiden University Medical Center, Leiden, the Netherlands. Electronic address: f.j.t.staal@lumc.nl., Pike-Overzet K; Leiden University Medical Center, Leiden, the Netherlands., de Kivit S; Leiden University Medical Center, Leiden, the Netherlands., Ott de Bruin L; Leiden University Medical Center, Leiden, the Netherlands., Mamede L; International Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium., Pergent M; International Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium., Prevot J; International Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium., Rothe M; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Schambach A; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Lankester A; Leiden University Medical Center, Leiden, the Netherlands.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 May 07; Vol. 33 (5), pp. 1869-1870. Date of Electronic Publication: 2025 Mar 18.
Publication Type: Letter
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE; In Process
Database: MEDLINE Ultimate
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PubType: Editorial & Opinion
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  Data: Safety and efficacy of gene therapy for RAG1-deficient SCID.
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  Data: <searchLink fieldCode="AU" term="%22Staal+FJT%22">Staal FJT</searchLink>; Leiden University Medical Center, Leiden, the Netherlands. Electronic address: f.j.t.staal@lumc.nl.<br /><searchLink fieldCode="AU" term="%22Pike-Overzet+K%22">Pike-Overzet K</searchLink>; Leiden University Medical Center, Leiden, the Netherlands.<br /><searchLink fieldCode="AU" term="%22de+Kivit+S%22">de Kivit S</searchLink>; Leiden University Medical Center, Leiden, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Ott+de+Bruin+L%22">Ott de Bruin L</searchLink>; Leiden University Medical Center, Leiden, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Mamede+L%22">Mamede L</searchLink>; International Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium.<br /><searchLink fieldCode="AU" term="%22Pergent+M%22">Pergent M</searchLink>; International Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium.<br /><searchLink fieldCode="AU" term="%22Prevot+J%22">Prevot J</searchLink>; International Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium.<br /><searchLink fieldCode="AU" term="%22Rothe+M%22">Rothe M</searchLink>; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Schambach+A%22">Schambach A</searchLink>; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Lankester+A%22">Lankester A</searchLink>; Leiden University Medical Center, Leiden, the Netherlands.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2025 May 07; Vol. 33 (5), pp. 1869-1870. <i>Date of Electronic Publication: </i>2025 Mar 18.
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  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>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE; In Process
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        Value: 10.1016/j.ymthe.2025.02.044
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      – Code: eng
        Text: English
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        StartPage: 1869
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      – TitleFull: Safety and efficacy of gene therapy for RAG1-deficient SCID.
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            – D: 07
              M: 05
              Text: 2025 May 07
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