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)] | 15 Claims |
1. A method of treating cone-rod dystrophy type 6 (CORD6) in a subject, the method comprising injecting into an eye of the subject having CORD6:
a first nucleic acid that encodes a guide RNA (gRNA) that targets both an autosomal dominant allele of a guanylate cyclase 2D (GUCY2D) gene and a wild-type allele of a GUCY2D gene in the subject,
a second nucleic acid that encodes an RNA-guided endonuclease, and
a replacement nucleic acid that comprises a functional GUCY2D gene that is hardened such that the gRNA no longer recognizes a target site in the gene, or the RNA-guided endonuclease no longer recognizes the protospacer adjacent motif (PAM) site adjacent to the target site.
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