Erik J. Sontheimer,Raed Ibraheim,Wen Xue,Aamir Mir,Alireza Edraki,Ildar Gainetdinov
申请号：
US16186352
公开号：
US20190338308A1
申请日:
2018.11.09
申请国别(地区):
US
年份:
2019
代理人:
摘要：
The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hyperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide RNA sequences that permit a complete sgRNA-Nme1Cas9 vector to be inserted into an adeno-associated viral plasmid that is compatible for in vivo administration. Furthermore, Type II-C Cas9 orthologs have been identified that target protospacer adjacent motif sequences limited to between one-four required nucleotides.
