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News and Views
Nature 435, 577-579 (2 June 2005) | doi:10.1038/435577a; Published online 1 June 2005
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Gene therapy: The moving finger
Katherine A. High1
Abstract
DNA-cleaving enzymes trigger a repair process that can now be harnessed to correct mutations in the human genome in vitro. This represents another step towards gene-correction strategies for treating human disease.
Genetic engineering and gene-transfer technologies have produced a wealth of new ideas about how to treat genetic diseases. Many of these have been applied quite successfully in cultured cells and even in mouse models of human disease, but have proven remarkably difficult to translate into clinical practice.
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Katherine A. High is at The Children's Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania
19104, USA.
Email: high@email.chop.edu
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