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US 2026/0144897 A1Published application
Methods of Targeting Mutant Cells
AssigneePresident and Fellows of Harvard College
InventorsMarkus Basan, Avik Mukherjee
Life of this patent
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Abstract
The present disclosure provides methods and systems for targeting cell with mutations.
Claims34
A method of treating a collection of cells comprising (1) a target mutated cell including a first mutation in cellular DNA and (2) a plurality of cells lacking the first mutation comprising
- (a) administering to the collection of cells (1) a nucleic acid construct comprising
- (i) a first nucleic acid encoding a treatment molecule wherein the nucleic acid construct optionally lacks one or more expression elements, such as for stable expression before integration of the construct and optionally targets the first mutation, and
- (ii) optionally a second nucleic acid encoding a gene editor, wherein the gene editor is expressed, wherein the gene editor optionally targets the first mutation and cleaves the cellular DNA at a target integration site,
- wherein at least either the nucleic acid construct or the gene editor targets the first mutation,
- (b) integrating the first nucleic acid encoding the treatment molecule into the target integration site of the cellular DNA of the target mutated cell, and
- (c) expressing the first nucleic acid encoding the treatment molecule after integration, wherein the treatment molecule treats the target mutated cell.
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A system for targeting mutations in cancer cells comprising
- a first nucleic acid encoding a treatment molecule wherein the first nucleic acid optionally targets the first mutation, and a second nucleic acid encoding a gene editor,
- wherein the gene editor optionally targets the first mutation, and wherein at least either the first nucleic acid or the gene editor targets the first mutation.
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Dependencies are inferred from the claim text.