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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