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AssigneeScribe Therapeutics Inc.
InventorsBenjamin OAKES, Sean HIGGINS, Hannah SPINNER, Sarah DENNY, Brett T. STAAHL, Kian TAYLOR, Katherine BANEY, Isabel COLIN, Maroof ADIL, Cole URNES
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Provided herein are CasX:gNA systems comprising CasX polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a SOD1 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the SOD1 protein or the SOD1 regulatory element. Also provided are methods of using such CasX:gNA systems to modify cells having such mutations and utility in methods of treatment of a subject with a SOD1-related disease.
1-168. (canceled)
A composition comprising a CasX variant protein comprising a sequence selected from the group consisting of the sequences of SEQ ID NOS:
A composition comprising a CasX variant thereof and a first guide nucleic acid (gNA), wherein the gNA has a scaffold comprising a sequence having at least 70% sequence identity to a sequence selected from the group consisting of the sequences of SEQ ID NOS:
A vector:
A method of treating a SOD1-related disease in a subject in need thereof, comprising modifying a SOD1 gene having one or more mutations in a central nervous system (CNS) or peripheral nervous system (PNS) cell of the subject, the modifying comprising contacting said CNS or PNS cell with a therapeutically effective dose of a composition comprising a CasX variant protein comprising a sequence selected from the group consisting of the sequences of SEQ ID NOS:
Dependencies are inferred from the claim text.