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:
Jul. 08, 2025

Filed:

May. 03, 2019
Applicant:

University of Massachusetts, Boston, MA (US);

Inventors:

Scot A Wolfe, Winchester, MA (US);

Charles P Emerson, Jr., Lyndon, VT (US);

Sukanya Iyer, Marlborough, MA (US);

Sneha Suresh, Dracut, MA (US);

Christian Mueller, Concord, MA (US);

Jennifer Chen, Kingston, CA;

Dongsheng Guo, Shrewsbury, MA (US);

Oliver King, Somerville, MA (US);

Assignee:

UNIVERSITY OF MASSACHUSETTS, Westborough, MA (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 9/22 (2006.01); A61K 35/76 (2015.01); A61K 38/00 (2006.01);
U.S. Cl.
CPC ...
C12N 9/22 (2013.01); A61K 35/76 (2013.01); A61K 38/00 (2013.01); C12N 2310/20 (2017.05); C12N 2800/80 (2013.01);
Abstract

The present invention is directed to the filed of gene therapy. In particular, compositions and methods are disclosed that repair gene microduplication mutations by reversion to a wild type sequence. For example, the creation of a double stranded break by a programmable nuclease protein within a microduplication induces the microhomology mediated end joining DNA repair pathway that in the process of DNA repair removes the microduplication mutation and restores the wild type sequence.


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