发明名称 METHODS FOR MODULATING GENOME EDITING
摘要 Provided herein are methods and kits for modulating genome editing of target DNA. The invention includes using small molecules that enhance or repress homologydirected repair (HDR) and/or nonhomologous end joining (NHEJ) repair of double-strand breaks in a target DNA sequence. Also provided herein are methods for preventing or treating a genetic disease in a subject by enhancing precise genome editing to correct a mutation in a target gene associated with the genetic disease. Further provided herein are systems and methods for screening small molecule libraries to identify novel modulators of genome editing. The present invention can be used with any cell type and at any gene locus that is amenable to nuclease-mediated genome editing technology.
申请公布号 WO2016115326(A1) 申请公布日期 2016.07.21
申请号 WO2016US13375 申请日期 2016.01.14
申请人 THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY;THE J. DAVID GLADSTONE INSTITUTES, A TESTAMENTARYTRUST ESTABLISHED UNDER THE WILL OF J. DAVID GLADSTONE;THE REGENTS OF THE UNIVERSITY OF CALIFORNIA 发明人 QI, LEI S.;DING, SHENG;YU, CHEN
分类号 C12N15/90;A61K38/46;C12Q1/44;C12Q1/68 主分类号 C12N15/90
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