Review Article | Published:

Refining strategies to translate genome editing to the clinic

Nature Medicine volume 23, pages 415423 (2017) | Download Citation

Abstract

Recent progress in developing programmable nucleases, such as zinc-finger nucleases, transcription activator–like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)–Cas nucleases, have paved the way for gene editing to enter clinical practice. This translation is a result of combining high nuclease activity with high specificity and successfully applying this technology in various preclinical disease models, including infectious disease, primary immunodeficiencies, hemoglobinopathies, hemophilia and muscular dystrophy. Several clinical gene-editing trials, both ex vivo and in vivo, have been initiated in the past 2 years, including studies that aim to knockout genes as well as to add therapeutic transgenes. Here we discuss the advances made in the gene-editing field in recent years, and specify priorities that need to be addressed to expand therapeutic genome editing to further disease entities.

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Acknowledgements

We thank all members of the laboratories for helpful discussion. Current research in our labs is funded by the Federal Ministry of Education and Research (IFB—01EO0803; iMACnet—01EK1602B; HBV-TALE—01DG15005), the German Research Foundation (SFB1160—TP17), the European Commission's Horizon 2020 Program (SCIDNET—666908; CARAT—667980), Casebia Therapeutics (CAU-950-20140702) and the German Academic Exchange Service.

Author information

Affiliations

  1. Institute for Cell and Gene Therapy, Medical Center—University of Freiburg, Freiburg, Germany.

    • Tatjana I Cornu
    • , Claudio Mussolino
    •  & Toni Cathomen
  2. Center for Chronic Immunodeficiency, Medical Center—University of Freiburg, Freiburg, Germany.

    • Tatjana I Cornu
    • , Claudio Mussolino
    •  & Toni Cathomen
  3. Faculty of Medicine, University of Freiburg, Freiburg, Germany.

    • Toni Cathomen

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Competing interests

T.C. is a consultant for TRACR Hematology.

Corresponding author

Correspondence to Toni Cathomen.

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DOI

https://doi.org/10.1038/nm.4313

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