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:
Jan. 31, 2023

Filed:

Aug. 02, 2017
Applicant:

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

Inventors:

Morgan Lee Maeder, Jamaican Plain, MA (US);

Rina J. Mepani, Belmont, CA (US);

Michael Stefanidakis, Brookline, MA (US);

Assignee:

EDITAS MEDICINE, INC., Cambridge, MA (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/90 (2006.01); A61K 35/761 (2015.01); A61K 9/00 (2006.01); C12N 15/11 (2006.01); C12N 9/22 (2006.01); C12N 15/113 (2010.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/907 (2013.01); A61K 9/0048 (2013.01); A61K 35/761 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 2310/20 (2017.05); C12N 2320/32 (2013.01); C12N 2330/50 (2013.01); C12N 2330/51 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/80 (2013.01);
Abstract

Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.


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