Journal
SCIENCE CHINA-LIFE SCIENCES
Volume 60, Issue 5, Pages 468-475Publisher
SCIENCE PRESS
DOI: 10.1007/s11427-017-9057-2
Keywords
CRISPR/Cas9; gene therapy; in vivo; ex vivo
Categories
Funding
- National Natural Science Foundation of China [31371455, 31171318, 81330049]
- Science and Technology Commission of Shanghai Municipality [14140900300]
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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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