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A blueprint to accelerate rare pediatric gene therapy approvals.

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Title: A blueprint to accelerate rare pediatric gene therapy approvals.
Authors: Abou-El-Enein M; USC/CHLA Cell Therapy Program, University of Southern California and Children's Hospital of Los Angeles, Los Angeles, CA, USA. abouelenein@med.usc.edu.; Division of Medical Oncology, Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. abouelenein@med.usc.edu.; Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. abouelenein@med.usc.edu.; Department of Regulatory and Quality Sciences, Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, CA, USA. abouelenein@med.usc.edu., Walters MC; Department of Pediatrics, UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA., Sanghavi D; Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA, USA., Kohn DB; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.; The Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA, USA.
Source: Nature medicine [Nat Med] 2026 Apr; Vol. 32 (4), pp. 1170-1171.
Publication Type: Letter
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE; In Process
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  Data: <searchLink fieldCode="AU" term="%22Abou-El-Enein+M%22">Abou-El-Enein M</searchLink>; USC/CHLA Cell Therapy Program, University of Southern California and Children's Hospital of Los Angeles, Los Angeles, CA, USA. abouelenein@med.usc.edu.; Division of Medical Oncology, Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. abouelenein@med.usc.edu.; Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. abouelenein@med.usc.edu.; Department of Regulatory and Quality Sciences, Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, CA, USA. abouelenein@med.usc.edu.<br /><searchLink fieldCode="AU" term="%22Walters+MC%22">Walters MC</searchLink>; Department of Pediatrics, UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.<br /><searchLink fieldCode="AU" term="%22Sanghavi+D%22">Sanghavi D</searchLink>; Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Kohn+DB%22">Kohn DB</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.; The Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA, USA.
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