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.
Claims65
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 comprises an amino acid sequence at least 95% identical to SEQ ID NO:
- 922 and comprises one or more mutations relative to SEQ ID NO:
- 922;
- (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.
A gene editing system for genetic editing of a hydroxyacid oxidase 1 (HAO1) gene, comprising
- (i) a Cas12i polypeptide or a first nucleic acid encoding the Cas12i polypeptide, optionally wherein the Cas12i polypeptide is a Cas12i2 polypeptide;
- (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 exon 1 or exon 2 of 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.
An RNA guide, comprising (i) a spacer sequence that is specific to a target sequence in a hydroxyacid oxidase 1 (HAO1) gene, wherein the target sequence is adjacent to a protospacer adjacent motif (PAM) comprising the motif of 5′-TTN-3′, which is located 5′ to the target sequence;
- and (ii) a direct repeat sequence.
Dependencies are inferred from the claim text.