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CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy

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Abstract

CRISPR/Cas9 is a versatile genome-editing tool which is widely used for modifying the genome of both prokaryotic and eukaryotic organisms for basic research and applications. An increasing number of reports have demonstrated that CRISPR/Cas9-mediated genome editing is a powerful technology for gene therapy. Here, we review the recent advances in CRISPR/Cas9-mediated gene therapy in animal models via different strategies and discuss the challenges as well as future prospects.

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Acknowledgements

This work was supported by the National Natural Science Foundation of China (31371455, 31171318 to Dali Li, 81330049 to Mingyao Liu), the Science and Technology Commission of Shanghai Municipality (14140900300 to Dali Li).

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  1. Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, 200241, China

    Xiaohui Zhang, Liren Wang, Mingyao Liu & Dali Li

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  1. Xiaohui Zhang
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  2. Liren Wang
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  3. Mingyao Liu
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  4. Dali Li
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Correspondence to Mingyao Liu or Dali Li.

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Zhang, X., Wang, L., Liu, M. et al. CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy. Sci. China Life Sci. 60, 468–475 (2017). https://doi.org/10.1007/s11427-017-9057-2

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