Rational designs of in vivo CRISPR-Cas delivery systems

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 extraand intra-cellular barriers, therefore largely limiting the applications of CRISPR-based therapeutics in vivo. In this review, we summarize the features of plasmid-, RNAand ribonucleoprotein (RNP)-based CRISPR-Cas therapeutics. Then, we survey the current in vivo delivery systems. We specify the requirements for efficient in vivo delivery in clinical settings, and highlight both efficiency and safety for different CRISPR-Cas tools. (c) 2019 Published by Elsevier B.V.

Automated summary

The CRISPR-Cas system has revolutionized genome editing, but efficient delivery to target tissues/cells remains a challenge. This review summarizes the characteristics of different CRISPR-Cas therapeutic tools and investigates current in vivo delivery systems, emphasizing the requirements for efficient delivery in clinical settings and the safety of different tools.