MULTILAYER GENETIC SAFETY KILL CIRCUITS BASED ON SINGLE CAS9 PROTEIN AND MULTIPLE ENGINEERED GRNA IN MAMMALIAN CELLS

摘要

Aspects of the disclosure relate to synthetic regulatory systems composed of a multifunctional Cas [clustered regularly interspaced short palindromic repeat (CRISPR)-associated (Cas)] nuclease and at least two distinct guide RNAs (gRNAs). The synthetic regulatory system modulates cleavage and transcription, including repression and activation, in a mammalian cell such as a human cell.