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

Affiliations

¹ Department of Neurology, Biochemistry and Division of Medical Genetics, University of Washington, Seattle, WA, USA. Electronic address: jsc5@uw.edu.
² 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.
³ 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.
⁴ Powell Gene Therapy Center at the University of Florida, Gainesville, FL, USA.
⁵ American Society of Gene & Cell Therapy, Waukesha, WI, USA.

Jeffrey S Chamberlain et al. Mol Ther. 2025.

. 2025 Oct 1;33(10):4694-4695.

doi: 10.1016/j.ymthe.2025.08.029. Epub 2025 Sep 12.

¹ Department of Neurology, Biochemistry and Division of Medical Genetics, University of Washington, Seattle, WA, USA. Electronic address: jsc5@uw.edu.
² 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.
³ 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.
⁴ Powell Gene Therapy Center at the University of Florida, Gainesville, FL, USA.
⁵ American Society of Gene & Cell Therapy, Waukesha, WI, USA.

No abstract available

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