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Commentary

Nature 427, 779-781 (26 February 2004) | doi:10.1038/427779a

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The future of gene therapy

Marina Cavazzana-Calvo, Adrian Thrasher & Fulvio Mavilio

  1. Marina Cavazzana-Calvo is at the Immunology and Pediatric Haematology Unit, Hospital Necker, 75743 Paris, Cedex 15, France.
  2. Adrian Thrasher is at the Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.
  3. Fulvio Mavilio is at the Istituto Scientifico H. San Raffaele, Via Olgettina 58, 20132 Milano, and in the Department of Biomedical Sciences, University of Modena, Via Campi 287, 41100 Modena, Italy.

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Balancing the risks and benefits of clinical trial.

Gene therapy has the potential to treat devastating inherited diseases for which there is little hope of finding a conventional cure. In the late 1990s, our groups in Paris, London and Milan began treating children suffering from rare immune disorders (severe combined immunodeficiencies, or SCIDs).

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