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Gene therapy

The moving finger

Nature volume 435, pages 577–579 (2005)Cite this article

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. On page 646 of this issue, however, Urnov and colleagues1 report a new strategy that combines two biological processes that are highly evolutionarily conserved to correct a genetic mutation in human cells. Their success rate is theoretically high enough to result in clinical benefit. Their findings, building on and extending previous studies of this strategy2,3, are a considerable step towards a successful genetic-engineering approach to treating human disease.

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Figure 1: Gene correction.

References

  1. Urnov, F. D. et al. Nature 435, 646–651 (2005).

    ADS  CAS  Article  Google Scholar 

  2. Porteus, M. H. & Baltimore, D. Science 300, 763 (2003).

    Article  Google Scholar 

  3. Bibikova, M. et al. Science 300, 764 (2003).

    CAS  Article  Google Scholar 

  4. Kim, Y. G., Cha, J. & Chandrasegaran, S. Proc. Natl Acad. Sci. USA 93, 1156–1160 (1996).

    ADS  CAS  Article  Google Scholar 

  5. Tupler, R. et al. Nature 409, 832–833 (2001).

    ADS  CAS  Article  Google Scholar 

  6. Klug, A. FEBS Lett. 579, 892–894 (2005).

    CAS  Article  Google Scholar 

  7. Cavazzana-Calvo, M. et al. Science 288, 669–672 (2000).

    ADS  CAS  Article  Google Scholar 

  8. Hacein-Bey-Abina, S. et al. N. Engl. J. Med. 348, 255–256 (2003).

    Article  Google Scholar 

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Authors and Affiliations

  1. The Children's Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, 19104, Pennsylvania, USA

    Katherine A. High

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  1. Katherine A. High
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High, K. The moving finger. Nature 435, 577–579 (2005). https://doi.org/10.1038/435577a

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  • DOI: https://doi.org/10.1038/435577a

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