The Responsible Development of Adeno-Associated Virus Gene Therapies
Several unexpected fatalities in patients who received adeno-associated virus (AAV)-based gene therapies have recently occurred. These tragic events have cast a pall over the entire sector with some stakeholders suggesting that AAV is patently unsafe as a gene delivery platform and ought not to be pursued. This conclusion is not warranted.
Authors
- James M. Wilson (ORCID: https://orcid.org/0000-0002-9630-3131)
- Arthur L. Caplan (ORCID: https://orcid.org/0000-0002-4061-8011)
Institutions
- Integrated BioTherapeutics (United States) (US)
- New York University (US)
Publication Details
- Journal
- Human Gene Therapy
- Published
- 2025-09-30
- DOI
- https://doi.org/10.1177/10430342251359668
- Citations
- 1
- Primary Topic
- CAR-T cell therapy research
- Type
- article
- Field-Weighted Citation Impact
- 1.05