[EN] QUINOXALINONE COMPOUNDS, COMPOSITIONS, METHODS, AND KITS FOR INCREASING GENOME EDITING EFFICIENCY<br/>[FR] COMPOSÉS DE QUINOXALINONE, COMPOSITIONS, PROCÉDÉS ET KITS POUR AUGMENTER L'EFFICACITÉ D'ÉDITION DU GÉNOME
申请人:VERTEX PHARMA
公开号:WO2019143677A1
公开(公告)日:2019-07-25
Compounds, 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 (DNA-PK) inhibitor disclosed herein. Kits and compositions for editing a target gene comprise a genome editing system and a DNA-PK inhibitor disclosed herein.
化合物,编辑目标基因组区域的方法,通过HDR途径修复DNA断裂的方法,通过NHEJ途径抑制或抑制修复DNA断裂的方法,以及修改基因或蛋白表达的方法包括向包括一个或多个目标基因组区域的一个或多个细胞施用本文披露的基因组编辑系统和DNA蛋白激酶(DNA-PK)抑制剂。用于编辑目标基因的试剂盒和组合物包括本文披露的基因组编辑系统和DNA-PK抑制剂。