	US 12,460,231 B2
	Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
Morgan L. Maeder, Jamaica Plain, MA (US); and David A. Bumcrot, Belmont, MA (US)
Assigned to EDITAS MEDICINE, INC., Cambridge, MA (US)
Appl. No. 15/300,991
Filed by EDITAS MEDICINE, INC., Cambridge, MA (US)
PCT Filed Apr. 1, 2015, PCT No. PCT/US2015/023906 § 371(c)(1), (2) Date Sep. 30, 2016, PCT Pub. No. WO2015/153780, PCT Pub. Date Oct. 8, 2015.
Claims priority of provisional application 61/974,327, filed on Apr. 2, 2014.
Prior Publication US 2017/0029850 A1, Feb. 2, 2017
Int. Cl. C12N 15/90 (2006.01); A61K 38/46 (2006.01); A61K 47/54 (2017.01); A61K 48/00 (2006.01); C12N 9/22 (2006.01); C12N 15/10 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01)

CPC C12N 15/907 (2013.01) [A61K 38/465 (2013.01); A61K 47/549 (2017.08); A61K 48/00 (2013.01); C12N 9/22 (2013.01); C12N 15/102 (2013.01); C12N 15/11 (2013.01); C12N 15/113 (2013.01); C12N 2310/10 (2013.01); C12N 2310/20 (2017.05); C12N 2320/30 (2013.01)]

11 Claims

1. A method of altering a cell comprising contacting the cell with:

(a) a first guide (gRNA) molecule comprising a first targeting domain which is complementary with a first target domain from the MYOC gene, wherein the first target domain is located within 500 bp of a start codon of the MYOC gene, wherein the first targeting domain is configured to provide a double strand break in a region of the MYOC gene which is complementary to a sequence that is the same as, or differs by no more than 3 nucleotides from, a nucleic acid sequence of SEQ ID NO:499 in the presence of a Cas9 molecule, and wherein the double strand break results in knockout of the MYOC gene; and

(b) the Cas9 molecule.