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US 11,821,012 B2Granted
Gene editing systems comprising an RNA guide targeting hydroxyacid oxidase 1 (HAO1) and uses thereof
AssigneeArbor Biotechnologies, Inc.
InventorsQuinton Norman Wessells, Jeffrey Raymond Haswell, Tia Marie Ditommaso, Noah Michael Jakimo, Sejuti Sengupta
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Abstract
Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.
Claims22
A gene editing system for genetic editing of a hydroxyacid oxidase 1 (HAO1) 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:
- 922, and wherein, relative to SEQ ID NO:922, the Cas12i2 polypeptide comprises mutations at positions D581, I926, and V1030;
- (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 HAO1 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.
Dependencies are inferred from the claim text.