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1. (WO2017040786) MULTILAYER GENETIC SAFETY KILL CIRCUITS BASED ON SINGLE CAS9 PROTEIN AND MULTIPLE ENGINEERED GRNA IN MAMMALIAN CELLS

Pub. No.:    WO/2017/040786    International Application No.:    PCT/US2016/049907
Publication Date: Fri Mar 10 00:59:59 CET 2017 International Filing Date: Fri Sep 02 01:59:59 CEST 2016
IPC: A61K 48/00
C12N 15/00
C12N 15/87
C07H 21/04
Applicants: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Inventors: KIANI, Samira
WEISS, Ron
EBRAHIMKHANI, Mohammad, Reza
Title: MULTILAYER GENETIC SAFETY KILL CIRCUITS BASED ON SINGLE CAS9 PROTEIN AND MULTIPLE ENGINEERED GRNA IN MAMMALIAN CELLS
Abstract:
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.