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1. (WO2018013840) METHODS, COMPOSITIONS AND KITS FOR INCREASING GENOME EDITING EFFICIENCY

Pub. No.:    WO/2018/013840    International Application No.:    PCT/US2017/041979
Publication Date: Fri Jan 19 00:59:59 CET 2018 International Filing Date: Fri Jul 14 01:59:59 CEST 2017
IPC: C07D 401/14
C12N 15/10
Applicants: VERTEX PHARMACEUTICALS INCORPORATED
Inventors: ABDUL-MANAN, Norzehan
NEWSOME, David A.
ZWAHLEN, Jacque
Title: METHODS, COMPOSITIONS AND KITS FOR INCREASING GENOME EDITING EFFICIENCY
Abstract:
Methods of editing a target genomic region(s), methods of repairing of a DNA break via a HDR pathway, methods of inhibiting or suppressing repair of a DNA break via a NHEJ pathway, and methods of modifying expression of a gene(s) or protein(s) comprise administering to one or more cells that include one or more target genomic regions, a genome editing system and a DNA protein-kinase (DNAPK) inhibitor disclosed herein. Kits and compositions for editing a target gene comprise a genome editing system and a DNAPK inhibitor disclosed herein.