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:
Aug. 22, 2017

Filed:

Aug. 18, 2014
Applicant:

President and Fellows of Harvard College, Cambridge, MA (US);

Inventors:

David R. Liu, Lexington, MA (US);

John Anthony Zuris, Cambridge, MA (US);

David B. Thompson, Cambridge, MA (US);

Assignee:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 9/127 (2006.01); A61K 47/12 (2006.01); A61K 38/45 (2006.01); A61K 38/46 (2006.01); C12N 15/85 (2006.01); C12N 9/12 (2006.01); C12N 9/96 (2006.01); C12N 9/22 (2006.01); C07K 14/195 (2006.01); C07K 14/435 (2006.01); C07K 14/46 (2006.01); C07K 14/47 (2006.01); A61K 38/00 (2006.01); A61K 48/00 (2006.01); A61K 38/17 (2006.01); C12N 9/14 (2006.01); C07K 14/00 (2006.01);
U.S. Cl.
CPC ...
A61K 47/12 (2013.01); A61K 9/127 (2013.01); A61K 38/1767 (2013.01); A61K 38/45 (2013.01); A61K 38/465 (2013.01); C07K 14/00 (2013.01); C07K 14/195 (2013.01); C07K 14/43595 (2013.01); C07K 14/463 (2013.01); C07K 14/4702 (2013.01); C12N 9/1241 (2013.01); C12N 9/14 (2013.01); C12N 9/22 (2013.01); C12N 9/96 (2013.01); C07K 2319/60 (2013.01); C07K 2319/80 (2013.01); C07K 2319/85 (2013.01); C12Y 207/07 (2013.01); C12Y 301/00 (2013.01);
Abstract

Compositions, methods, strategies, kits, and systems for the supercharged protein-mediated delivery of functional effector proteins into cells in vivo, ex vivo, or in vitro are provided. Compositions, methods, strategies, kits, and systems for delivery of functional effector proteins using cationic lipids and cationic polymers are also provided. Functional effector proteins include, without limitation, transcriptional modulators (e.g., repressors or activators), recombinases, nucleases (e.g., RNA-programmable nucleases, such as Cas9 proteins; TALE nuclease, and zinc finger nucleases), deaminases, and other gene modifying/editing enzymes. Functional effector proteins include TALE effector proteins, e.g., TALE transcriptional activators and repressors, as well as TALE nucleases. Compositions, methods, strategies, and systems for the delivery of functional effector proteins into cells is useful for therapeutic and research purposes, including, but not limited to, the targeted manipulation of a gene associated with disease, the modulation of the expression level of a gene associated with disease, and the programming of cell fate.


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