26 February 2004
Gene therapy: No risk, no gain
Retroviruses are the only clinical tool currently available to introduce a permanent genetic modification into stem cells, and to treat life-threatening conditions like severe combined immunodeficiencies (SCIDs). The successful use of such therapy in SCID patients was greeted with great excitement just four years ago. But euphoria soon turned to despair when two of the 10 children treated in France developed leukaemia-like conditions. In this week's Commentary, Marina Cavazzana-Calvo, Adrian Thrasher and Fulvio Mavilio argue that, with little hope of finding a conventional cure for these devastating inherited diseases, it is vital to find a workable balance between feasibility, safety and efficacy for gene therapy. By rethinking guidelines on dosage and other variables, and introducing sophisticated molecular monitoring of the progress of treatments, it may yet be possible to restore confidence in the future of gene therapy based on retroviral vectors.
The future of gene therapy
MARINA CAVAZZANA-CALVO, ADRIAN THRASHER & FULVIO
MAVILIO
Balancing the risks and benefits of clinical trial.
Nature 427, 779–781 (2004); doi:10.1038/427779a
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