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专利名称：DELIVERY, USE AND THERAPEUTIC
APPLICATIONS OF THE CRISPR-CASSYSTEMS AND COMPOSITIONS FOR HBVAND VIRAL DISEASES AND DISORDERS
发明人：BHATIA, Sangeeta,RICE, Charles,ZHANG,
Feng,COX, David Benjamin Turitz,RAMANAN,Vyas,SCHWARTZ, Robert,SHLOMAI, Amir
申请号：EP14830911.5申请日：20141212公开号：EP3080261B1公开日：20190522
摘要：The invention provides for delivery, engineering and optimization of systems,methods, and compositions for manipulation of sequences and/or activities of targetsequences. Provided are delivery systems and tissues or organ which are targeted assites for delivery. Also provided are vectors and vector systems some of which encodeone or more components of a CRISPR complex, as well as methods for the design anduse of such vectors. Also provided are methods of directing CRISPR complex formation ineukaryotic cells to ensure enhanced specificity for target recognition and avoidance oftoxicity and to edit or modify a target site in a genomic locus of interest to alter orimprove the status of a disease or a condition.
申请人：BROAD INST INC,MASSACHUSETTS INST TECHNOLOGY,UNIV ROCKEFELLER
地址：US,US,US
国籍：US,US,US
代理机构：Icely, Dominic Michael
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