CRISPR-BASED GENOME MODIFICATION AND REGULATION

摘要

The present invention provides RNA-guided endonucleases, which are engineered for expression in eukaryotic cells or embryos, and methods of using the RNA-guided endonuclease for targeted genome modification in in eukaryotic cells or embryos. Also provided are fusion proteins, wherein each fusion protein comprises a CRISPR/Cas-like protein or fragment thereof and an effector domain. The effector domain can be a cleavage domain, an epigenetic modification domain, a transcriptional activation domain, or a transcriptional repressor domain. Also provided are methods for using the fusion proteins to modify a chromosomal sequence or regulate expression of a chromosomal sequence.

主权项

1. An isolated endonuclease comprising at least one nuclear localization signal, at least one nuclease domain, and at least one domain that interacts with a guide RNA to guide the endonuclease to a target site in a chromosomal sequence for cleavage.