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The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve.

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Title: The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve.
Authors: Chamberlain JS; Department of Neurology, Biochemistry and Division of Medical Genetics, University of Washington, Seattle, WA, USA. Electronic address: jsc5@uw.edu., Davidson BL; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; University of Pennsylvania, Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address: davidsonbl@chop.edu., George LA; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; University of Pennsylvania, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Byrne BJ; Powell Gene Therapy Center at the University of Florida, Gainesville, FL, USA., Barrett D; American Society of Gene & Cell Therapy, Waukesha, WI, USA.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Oct 01; Vol. 33 (10), pp. 4694-4695. Date of Electronic Publication: 2025 Sep 12.
Publication Type: Journal Article
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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  Data: The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve.
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  Data: <searchLink fieldCode="AU" term="%22Chamberlain+JS%22">Chamberlain JS</searchLink>; Department of Neurology, Biochemistry and Division of Medical Genetics, University of Washington, Seattle, WA, USA. Electronic address: jsc5@uw.edu.<br /><searchLink fieldCode="AU" term="%22Davidson+BL%22">Davidson BL</searchLink>; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; University of Pennsylvania, Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address: davidsonbl@chop.edu.<br /><searchLink fieldCode="AU" term="%22George+LA%22">George LA</searchLink>; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; University of Pennsylvania, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Byrne+BJ%22">Byrne BJ</searchLink>; Powell Gene Therapy Center at the University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Barrett+D%22">Barrett D</searchLink>; American Society of Gene & Cell Therapy, Waukesha, WI, USA.
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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 Oct 01; Vol. 33 (10), pp. 4694-4695. <i>Date of Electronic Publication: </i>2025 Sep 12.
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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.2025.08.029
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