Massachusetts Institute of Technology;The Broad Institute Inc.
发明人:
Feng ZHANG,Matthias HEIDENREICH,Lukasz SWIECH
申请号：
US14971169
公开号：
US20160153004A1
申请日:
2015.12.16
申请国别(地区):
US
年份:
2016
代理人:
摘要：
The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which include post mitotic cells which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
