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:
Nov. 07, 2023

Filed:

Aug. 28, 2018
Applicants:

University of Florida Research Foundation, Incorporated, Gainesville, FL (US);

Editas Medicine, Inc., Cambridge, MA (US);

Inventors:

Shannon E. Boye, Gainesville, FL (US);

Sanford L. Boye, Gainesville, FL (US);

Morgan Maeder, Jamaica Plain, MA (US);

Assignees:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/79 (2006.01); C07H 21/04 (2006.01); A61K 35/761 (2015.01); C12N 15/86 (2006.01); C07H 21/02 (2006.01);
U.S. Cl.
CPC ...
A61K 48/005 (2013.01); A61K 35/761 (2013.01); A61K 48/0075 (2013.01); C12N 15/86 (2013.01); C07H 21/02 (2013.01); C07H 21/04 (2013.01); C12N 2310/20 (2017.05); C12N 2750/14143 (2013.01); C12N 2800/80 (2013.01);
Abstract

Provided herein are methods and compositions for treating an eye disorder, for example cone-rod dystrophy type 6 (CORD6). In certain aspects, a therapeutically effective amount of a composition comprising nucleic acids is administered to a subject to treat an autosomal dominant disorder or condition, such as a condition associated with a dominant mutation in a guanylate cyclase 2D (GUCY2D) gene, such as knocking out a dominant mutant form of the gene in the subject. Further provided herein are recombinant AAV particles that comprise one or more recombinant AAV genomes comprising nucleic acids that encode a guide RNA that targets a GUCY2D gene and/or an RNA-guided endonuclease.


Find Patent Forward Citations

Loading…