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Progress and problems with the use of viral vectors for gene therapy

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NATURE REVIEWS GENETICS
卷 4, 期 5, 页码 346-358

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NATURE PUBLISHING GROUP
DOI: 10.1038/nrg1066

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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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