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

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SCIENCE CHINA Life Sciences, Volume 60, Issue 5: 468-475(2017) https://doi.org/10.1007/s11427-017-9057-2

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

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  • ReceivedNov 29, 2016
  • AcceptedFeb 16, 2017
  • PublishedApr 20, 2017

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.


Funded by

Science and Technology Commission of Shanghai Municipality(14140900300 to Dali Li)

National Natural Science Foundation of China(31371455)


Acknowledgment

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).


Interest statement

The author(s) declare that they have no conflict of interest.


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