| 초록 |
The CRISPR-Cas system is a highly robust platform for genome engineering, attracting great interest in therapeutic applications. A single guide RNA recognizes the target sequence by complementary base pairing, allowing Cas9 to induce double-strand breaks within DNA. Despite the tremendous advances in applications of the CRISPR system, there are many challenges that need to be addressed for use as a therapeutic. Direct delivery of the ribonucleoprotein (RNP) complex has great advantages, such as reduced off-target effects and low immunogenicity, providing wide application potentials as a therapeutic in the clinic. In this study, we introduce click chemistry to produce stealth CRISPR nanocomplexes for efficient delivery and genome editing in vivo. Cas9 was derivatized with cationic polymer and sgRNA was PEGylated by copper free click chemistry as RNP complexes. We hope that the current approach could be applied in the clinic to treat various diseases such as cancer and genetic disorders. |
