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Published on: February 17, 2026
Progress and problems with the use of viral vectors for gene therapy
Clare E Thomas1, Anja Ehrhardt, Mark A Kay
1Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.
Abstract:
Gene therapy has a history of controversy. Encouraging results are starting to emerge from the clinic, but questions are still being asked about the safety of this new molecular medicine. With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
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