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US 2024/0181084 A1Published application
Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas Fusion Protein and Methods of Treatment
AssigneeUniversity of Rochester
InventorDouglas Matthew Anderson
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Abstract
The present disclosure provides proteins, nucleic acids, systems and methods for editing genomic material and method of treatment.
Claims38
A method of treating Friedreich's Ataxia in a subject, the method comprising administering:
- a) a fusion protein comprising a retroviral integrase (IN) or a fragment thereof, a CRISPR-associated (Cas) protein, and a nuclear localization signal (NLS) or a nucleic acid molecule comprising one or more nucleic acid sequences encoding a retroviral integrase (IN) or a fragment thereof, a CRISPR-associated (Cas) protein, and a nuclear localization signal (NLS);
- b) a guide nucleic acid comprising a targeting nucleotide sequence complimentary to a target region in the genome of the subject;
- c) a donor template nucleic acid comprising a U3 sequence, a U5 sequence and a donor template sequence, wherein the donor template sequence comprises a nucleic acid sequence encoding frataxin.
A fusion protein comprising:
- a) a CRISPR-associated (Cas) 14 protein;
- and
- b) a nuclear localization signal (NLS).
A system for editing genetic material, comprising in one or more vectors:
- a) a nucleic acid sequence encoding a fusion protein comprising a CRISPR-associated (Cas) 14 protein, and a nuclear localization signal (NLS);
- b) a nucleic acid sequence coding a CRISPR-Cas system guide RNA;
- and
- c) a nucleic acid sequence coding a donor template nucleic acid, wherein the donor template nucleic acid comprises a U3 sequence, a U5 sequence and a donor template sequence.
1 more element
Dependencies are inferred from the claim text.