US 12,234,449 B2
CRISPR/Cas-related methods and compositions for treating Leber's congenital amaurosis 10 (LCA10)
Morgan Lee Maeder, Jamaica Plain, MA (US); David A. Bumcrot, Belmont, MA (US); and Shen Shen, Watertown, MA (US)
Assigned to EDITAS MEDICINE, INC., Cambridge, MA (US)
Filed by EDITAS MEDICINE, INC., Cambridge, MA (US)
Filed on Mar. 7, 2022, as Appl. No. 17/688,782.
Application 15/904,269 is a division of application No. 14/644,181, filed on Mar. 10, 2015, granted, now 9,938,521, issued on Apr. 10, 2018.
Application 17/688,782 is a continuation of application No. 16/279,941, filed on Feb. 19, 2019, granted, now 11,268,086.
Application 16/279,941 is a continuation of application No. 15/904,269, filed on Feb. 23, 2018, granted, now 10,253,312, issued on Apr. 9, 2019.
Claims priority of provisional application 62/036,576, filed on Aug. 12, 2014.
Claims priority of provisional application 61/950,733, filed on Mar. 10, 2014.
Prior Publication US 2023/0025279 A1, Jan. 26, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/10 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01); C12N 15/86 (2006.01)
CPC C12N 15/1024 (2013.01) [C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 2310/10 (2013.01); C12N 2310/20 (2017.05); C12N 2750/14132 (2013.01); C12N 2750/14142 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01); C12N 2750/14151 (2013.01)] 17 Claims
 
1. A guide RNA (gRNA) molecule comprising a targeting domain which is complementary with a target domain from the CEP290 gene.
 
4. A nucleic acid that comprises: (a) a sequence that encodes a first gRNA molecule comprising a first targeting domain that is complementary with a first target domain in the CEP290 gene.
 
14. A CRISPR/Cas system comprising
a first guide RNA (gRNA) molecule comprising a first targeting domain which is complementary with a first target domain from the CEP290 gene;
a Cas9 molecule, wherein the first gRNA molecule is configured to form a first complex with the Cas9 molecule.