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US 2023/0116223 A1Published application
Nuclease-scaffold composition delivery platform
OwnerPhilip ROCHEinventor
Life of this patent
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Abstract
Described herein are methods, compositions, and systems for gene editing using polynucleotide modifying enzymes that do not require the use of chemical transfection agents for entry into cells.
Claims144
A composition for modifying a gene comprising
- a cell recognition domain (CRD);
- an endosome escape domain (EE);
- and
- a polynucleotide-modifying enzyme domain (PMNE);
- wherein the endosome escape domain is covalently coupled to the cell recognition domain.
2 more elements
13-14. (canceled)
A vector comprising a nucleotide sequence encoding a cell recognition domain, an endosome escape domain, and a polynucleotide-modifying enzyme domain.
A method for inducing death of cells bearing an EML4-ALK fusion gene, comprising contacting to said cell a composition comprising:
- a protein having at least 80% identity to SEQ ID NO 77, or a variant thereof, and
- a guide RNA targeting ALK4.
A method for increasing cell resistance to HIV infection, comprising contacting to said cell a composition comprising:
- a protein having at least 80% identity to SEQ ID NO:
- 87, or a variant thereof, and
- a guide RNA targeting the CXCR4 locus.
Dependencies are inferred from the claim text.