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Genome Editing: A Tool For Research and Therapy: Targeted genome editing hits the clinic

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Targeted genome editing by engineered endonucleases allows the precise introduction of gene deletions and substitutions into the target genome. In 'Bench to Bedside', Keith Joung and his colleagues discuss how genome-editing technologies could be applied to engineer disease-associated somatic variation into human cell lines and disease models. This would allow the functional interpretation of such variants, which could then be applied to molecular diagnostics in the clinic. In 'Bedside to Bench', Angelo Lombardo and Luigi Naldini consider the potential applications of genome editing in the clinic, in which engineered endonucleases have been shown to be safe. Endonucleases could replace disease-associated genes with wild-type versions or be used to delete genes encoding receptors essential to viral host entry to prevent infection.

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Figure 1: Exploiting targeted gene editing for therapeutic applications.

Katie Vicari/Nature Publishing Group

References

  1. Tebas, P. et al. N. Engl. J. Med. 370, 901–910 (2014).

    Article  CAS  Google Scholar 

  2. Holt, N. et al. Nat. Biotechnol. 28, 839–847 (2010).

    Article  CAS  Google Scholar 

  3. Genovese, P. et al. Nature 510, 235–240 (2014).

    Article  CAS  Google Scholar 

  4. Ciccia, A. & Elledge, S.J. Mol. Cell 40, 179–204 (2010).

    Article  CAS  Google Scholar 

  5. Liu, R. et al. Cell 86, 367–377 (1996).

    Article  CAS  Google Scholar 

  6. Hütter, G. et al. N. Engl. J. Med. 360, 692–698 (2009).

    Article  Google Scholar 

  7. Lombardo, A. et al. Nat. Biotechnol. 25, 1298–1306 (2007).

    Article  CAS  Google Scholar 

  8. Lombardo, A. et al. Nat. Methods 8, 861–869 (2011).

    Article  CAS  Google Scholar 

  9. Fischer, A., Hacein-Bey-Abina, S. & Cavazzana-Calvo, M. Gene 525, 170–173 (2013).

    Article  CAS  Google Scholar 

  10. Beerman, I., Seita, J., Inlay, M.A., Weissman, I.L. & Rossi, D.J. Cell Stem Cell 15, 37–50 (2014).

    Article  CAS  Google Scholar 

  11. Gabriel, R. et al. Nat. Biotechnol. 29, 816–823 (2011).

    Article  CAS  Google Scholar 

  12. Kim, H. & Kim, J.S. Nat. Rev. Genet. 15, 321–334 (2014).

    Article  CAS  Google Scholar 

Download references

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Correspondence to Luigi Naldini.

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Competing interests

The authors declare that they receive some research funding from Sangamo BioSciences to perform targeted genome editing with ZFNs and that they are inventors on filed patent applications on targeted genome editing.

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Lombardo, A., Naldini, L. Genome Editing: A Tool For Research and Therapy: Targeted genome editing hits the clinic. Nat Med 20, 1101–1103 (2014). https://doi.org/10.1038/nm.3721

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