The Board of Trustees of the Leland Stanford Junior University;The J. David Gladstone Institutes, a Testamentary Trust established under the Will of J. David Glads;The Regents of the University of Cal
发明人:
Lei S. Qi,Sheng Ding,Chen Yu
申请号：
US15649304
公开号：
US20180016601A1
申请日:
2017.07.13
申请国别(地区):
US
年份:
2018
代理人:
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 homology-directed 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.
