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. |
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| 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 |
| ISSN: | 1525-0024 |
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| DOI: | 10.1016/j.ymthe.2025.08.029 |
