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Gene therapy

Gene-editing therapy for neurological disease

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Guide RNA-mediated CRISPR–Cas nucleases are a powerful technology for the engineering of mammalian genomes. CRISPR–Cas9-dependent editing of mutated genes that cause Huntington disease and fragile X syndrome was recently achieved in cell-based models, heralding the first step towards developing this technology into viable therapeutics for neurological diseases.

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Figure 1: CRISPR–Cas9-based gene editing.

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Acknowledgements

M.A.M. and D.W.C. receive salary support from the Ludwig Institute for Cancer Research.

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Authors and Affiliations

  1. Moira A. McMahon and Don W. Cleveland are at the Ludwig Institute and Department of Cellular and Molecular Medicine, University of California, San Diego, 9500 Gilman Drive, La Jolla, California 92093–0670, USA.,

    Moira A. McMahon & Don W. Cleveland

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  1. Moira A. McMahon
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  2. Don W. Cleveland
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Correspondence to Don W. Cleveland.

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Competing interests

D.W.C. has been a consultant for Ionis Pharmaceuticals since 2003. M.A.M. declares no competing interests.

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McMahon, M., Cleveland, D. Gene-editing therapy for neurological disease. Nat Rev Neurol 13, 7–9 (2017). https://doi.org/10.1038/nrneurol.2016.190

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