Rational designs of in vivo CRISPR-Cas delivery systems.
Rational designs of in vivo CRISPR-Cas delivery systems.
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DOI：
10.1016/j.addr.2019.11.005
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发表时间：
2019-11
影响因子：
16.1
通讯作者：
Cong‐Fei Xu;Guojun Chen;Yingli Luo;Yue Zhang;Gui Zhao;Zi-Dong Lu;A. Czarna;Zhen Gu;Jun Wang-Jun-Wa
中科院分区：
文献类型：
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作者：
Cong‐Fei Xu;Guojun Chen;Yingli Luo;Yue Zhang;Gui Zhao;Zi-Dong Lu;A. Czarna;Zhen Gu;Jun Wang-Jun-Wa
当CRISPR-CAS系统首次在哺乳动物细胞中展示成功时，它开启了基因组编辑的一场革命。今天，科学家能够使用基于CRISPR-CAS的工具轻松地编辑基因组、调节基因转录、设计转录后事件和成像核酸。然而，由于许多细胞外和细胞内的屏障，有效地将CRISPR-CA运送到靶组织/细胞仍然具有挑战性，因此在很大程度上限制了基于CRISPR的治疗在体内的应用。本文综述了以质粒、RNA和核糖核蛋白(RNP)为基础的CRISPR-Cas疗法的特点。然后，我们对目前的活体递送系统进行了综述。我们明确了临床环境下对活体递送效率的要求，并强调了不同CRISPR-CAS工具的效率和安全性。
The CRISPR-Cas system initiated a revolution in genome editing when it was, for the first time, demonstrated success in the mammalian cells. Today, scientists are able to readily edit genomes, regulate gene transcription, engineer posttranscriptional events, and image nucleic acids using CRISPR-Cas-based tools. However, to efficiently transport CRISPR-Cas into target tissues/cells remains challenging due to many extra- and intra-cellular barriers, therefore largely limiting the applications of CRISPR-based therapeuticsin vivo. In this review, we summarize the features of plasmid-, RNA- and ribonucleoprotein (RNP)-based CRISPR-Cas therapeutics. Then, we survey the currentin vivodelivery systems. We specify the requirements for efficientin vivodelivery in clinical settings, and highlight both efficiency and safety for different CRISPR-Cas tools.