Patent ID: 11939607
Assignee: ARBOR BIOTECHNOLOGIES, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A gene editing system for genetic editing of a lactate dehydrogenase A (LDHA) gene, comprising
(i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, wherein the Cas12i2 polypeptide is a variant of SEQ ID NO:1166, and wherein, relative to SEQ ID NO: 1166, the Cas12i2 polypeptide comprises mutations at positions comprising D581, 1926, and V1030; and wherein the mutations are amino acid substitutions of D581R, I926R, and V1030G, respectively;
(ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an LDHA gene, the target sequence being adjacent to a protospacer adjacent motif (PAM) comprising the motif of 5′-TTN-3′, which is located 5′ to the target sequence.