The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Sep. 09, 2025

Filed:

Dec. 01, 2020
Applicants:

The Broad Institute, Inc., Cambridge, MA (US);

Massachusetts Institute of Technology, Cambridge, MA (US);

University of Iowa Research Foundation, Iowa City, IA (US);

Inventors:

Beverly Davidson, Iowa City, IA (US);

Chie-Yu Lin, Cambridge, MA (US);

Edgardo Rodriguez, Iowa City, IA (US);

Feng Zhang, Cambridge, MA (US);

Assignees:
Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/63 (2006.01); A01K 67/0276 (2024.01); A61K 31/713 (2006.01); A61K 38/46 (2006.01); A61K 48/00 (2006.01); B82Y 5/00 (2011.01); C12N 7/00 (2006.01); C12N 9/22 (2006.01); C12N 15/10 (2006.01); C12N 15/113 (2010.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01);
U.S. Cl.
CPC ...
C12N 15/63 (2013.01); A01K 67/0276 (2013.01); A61K 31/713 (2013.01); A61K 38/465 (2013.01); A61K 48/005 (2013.01); C12N 7/00 (2013.01); C12N 9/22 (2013.01); C12N 15/102 (2013.01); C12N 15/1082 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 15/907 (2013.01); C12Y 301/00 (2013.01); A01K 2217/075 (2013.01); A01K 2227/105 (2013.01); A01K 2267/0318 (2013.01); B82Y 5/00 (2013.01); C12N 2740/15043 (2013.01); C12N 2750/14143 (2013.01);
Abstract

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying 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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