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:
Jun. 07, 2022

Filed:

Aug. 17, 2017
Applicants:

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

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

Inventors:

Feng Zhang, Cambridge, MA (US);

David Arthur Scott, Cambridge, MA (US);

Winston Xia Yan, Cambridge, MA (US);

Sourav Choudhury, Cambridge, MA (US);

Matthias Heidenreich, Cambridge, MA (US);

Assignees:

THE BROAD INSTITUTE, INC., Cambridge, MA (US);

MASSACHUSETTS INSTITUTE OF TECHNOLOGY, Cambridge, MA (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/90 (2006.01); A61K 9/127 (2006.01); A61K 9/51 (2006.01); C12N 7/00 (2006.01); C12N 15/113 (2010.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/907 (2013.01); A61K 9/1271 (2013.01); A61K 9/5184 (2013.01); C12N 7/00 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 2310/20 (2017.05); C12N 2310/32 (2013.01); C12N 2740/13042 (2013.01); C12N 2740/13043 (2013.01); C12N 2800/22 (2013.01);
Abstract

Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity. Thus, in another aspect certain embodiments disclosed herein are directed to delivery vectors, constructs, and methods of delivering larger genes for systemic delivery.


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