期刊
SCIENCE CHINA-MATERIALS
卷 60, 期 6, 页码 511-515出版社
SCIENCE PRESS
DOI: 10.1007/s40843-016-5154-4
关键词
CRISPR-Cas9; drug delivery; gene therapy; nanomedicine; genome editing
The CRISPR-Cas system, especially the type II CRISPR-Cas9 system from Streptococcuspyogenes, has rapidly emerged as a popular genome editing tool. The development of Cas9 derivatives further expanded the toolbox of CRISPR-Cas9 based genome editing kit. However, therapeutic translation of the CRISPR-Cas9 systemin vivo is severely impeded by the absence of an appropriate delivery carrier. The complexity and high molecular weight of the CRISPR-Cas9 system, together with the physiological barriers for nucleus targeted cargo transportation have made it a huge challenge for in vivo therapeutic CRISPR-Cas9 delivery. Currently, the main stream carriers for systemic delivery of CRISPR-Cas9 are viral based, such as adeno-associated virus. However, the safety concerns surrounding viral vectors call for the development of non-viral nanocarriers. In this review, we survey the recent advances in the development of non-viral delivery systems for CRISPR-Cas9. Challenges and future directions in this field are also discussed.
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