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US 2026/0092271 A1Published application
Methods and compositions to control gene using genome editing
AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsJesse Engreitz, Gabriella Martyn, Michael Montgomery, Benjamin Doughty, Harold Jones, Katherine Guo
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Abstract
Provided herein are methods for screening edited genomic sequences for their effect on expression of a target gene, and for designing a target sequence edit for introducing to a target sequence of a nucleic acid molecule. Also provided are compositions and cells including the edited sequences, and methods for their use in preventing or treating a disease.
Claims44
A method of screening edited genomic sequences for their effect on expression of a target gene, the method comprising:
- providing a population of cells, the genomes of the population of cells comprising a target sequence;
- introducing a plurality of different target sequence edits into the genomes of the population of cells;
- measuring a parameter for each cell of the population of cells, the parameter correlating with an expression level of the target gene;
- separating the population of cells into a plurality of sets based on the measured parameter;
- for each combination of (a) a set of the plurality of sets and (b) a target sequence edit of the plurality of different target sequence edits, determining a count of the target sequence edit incorporated into the genomes of the cells of the population of cells in the set;
- and
- for each target sequence edit of the plurality of different target sequence edits, based on the counts in the plurality of sets, computing an effect of the target sequence edit on the expression level of the target gene.
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Dependencies are inferred from the claim text.