Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

A composite, engineered technology, applied in other methods of inserting foreign genetic material, digestive system, respiratory diseases, etc.
CN110872583APending Publication Date: 2020-03-10THE BROAD INST INC +2
99 Cites 9 Cited by

Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
THE BROAD INST INC
Publication Date
2020-03-10

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Abstract

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 deliver 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 ceils 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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Description

[0001] This application is a subsection of an invention patent application with an application date of December 12, 2013, an application number of 2013800727522, and an invention title of "Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications" case application.

[0002] Related applications and cited references

[0003] This application claims benefit and priority to the following U.S. Provisional Patent Application Numbers: 61 / 736,527 filed December 12, 2012; 61 / 748,427 filed January 2, 2013; 61 / 758,468 filed January 30, 2013 , 61 / 769,046 filed February 25, 2013; 61 / 791,409 and 61 / 802,174 filed March 15, 2013, 61 / 806,375 filed March 28, 2013; 61 filed April 20, 2013 / 814,263; 61 / 819,803 filed May 6, 2013; 61 / 828,130 filed May 28, 2013; 61 / 835,931 and 61 / 836,123 filed June 17, 2013 and July 17, 2013 61 / 847,537.

[0004] Reference is also made to the following U.S. Provisional Patent Application Nos.:...

Claims

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