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Gene therapy progress and prospects: targeted gene repair

Gene Therapy volume 12, pages 639–646 (2005)Cite this article

Abstract

The capacity to correct a mutant gene within the context of the chromosome holds great promise as a therapy for inherited disorders but fulfilling this promise has proven to be challenging. However, steady progress is being made and the development of gene repair as a viable and robust approach is underway. Here, we present some of the recent advances that are helping to shape our thinking about the feasibility and the limitations of this technique. For the most part, these advances center on understanding the regulation of the reaction and validating its application in animal models.

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Acknowledgements

We are grateful to members of the community who communicated their unpublished results to us. We thank NIH and Tapestry Pharmaceuticals for continued financial support and we apologize to our colleagues whose work was not cited as primary references due only to space limitations. Hopefully, the cited reviews encompass their outstanding efforts.

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Authors and Affiliations

  1. Department of Biological Sciences, Delaware Biotechnology Institute, University of Delaware, Newark, DE, USA

    H Parekh-Olmedo, L Ferrara, E Brachman & E B Kmiec

  2. Department of Biology, Genetics, Biochemistry, University of Torino, Torino, Italy

    L Ferrara

  3. The Rockefeller University, New York, NY, 10021, USA

    E Brachman

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Parekh-Olmedo, H., Ferrara, L., Brachman, E. et al. Gene therapy progress and prospects: targeted gene repair. Gene Ther 12, 639–646 (2005). https://doi.org/10.1038/sj.gt.3302511

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