- 专利标题： Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
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申请号： US14971356申请日： 2015-12-16
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公开(公告)号： US10577630B2公开(公告)日： 2020-03-03
- 发明人: Feng Zhang , Le Cong , Fei Ran
- 申请人： The Broad Institute, Inc. , Massachusetts Institute of Technology , President and Fellows of Harvard College
- 申请人地址： US MA Cambridge US MA Cambridge US MA Cambridge
- 专利权人： THE BROAD INSTITUTE, INC.,MASSACHUSETTS INSTITUTE OF TECHNOLOGY,PRESIDENT AND FELLOWS OF HARVARD COLLEGE
- 当前专利权人： THE BROAD INSTITUTE, INC.,MASSACHUSETTS INSTITUTE OF TECHNOLOGY,PRESIDENT AND FELLOWS OF HARVARD COLLEGE
- 当前专利权人地址： US MA Cambridge US MA Cambridge US MA Cambridge
- 代理机构： Foley & Lardner LLP
- 代理商 Sunit Talapatra
- 主分类号： A01K67/00
- IPC分类号： A01K67/00 ; C12N15/90 ; C12N9/22 ; C12N15/63 ; C12N15/10 ; A01K67/027 ; C12N15/86 ; A61K48/00 ; C12N7/00
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 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.
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