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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Acknowledgements
M.A.M. and D.W.C. receive salary support from the Ludwig Institute for Cancer Research.
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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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DOI: https://doi.org/10.1038/nrneurol.2016.190
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