Genome editing enhancers
Assigneebluebird bio, Inc.
InventorsSasha ASTRAKHAN, Garrett C. HEFFNER
Life of this patent
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Abstract
The invention provides improved compositions for gene therapy compositions for treatment, prevention, or amelioration of numerous diseases, disorders, and conditions.
Claims118
A composition comprising a population of cells, a genome editing enhancer, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a population of cells, a genome editing enhancer, a donor repair template, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a genome editing enhancer, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a genome editing enhancer, a donor repair template, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a cell, an acridine, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a cell, an acridine, a donor repair template, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a cell, 9-aminoacridine, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising a cell, 9-aminoacridine, a donor repair template, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising an acridine, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising an acridine, a donor repair template, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising 9-aminoacridine, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A composition comprising 9-aminoacridine, a donor repair template, and an engineered nuclease, and/or optionally, an mRNA encoding the engineered nuclease.
A method of increasing genome editing in a population of cells comprising:
- (a) introducing an engineered nuclease into a population of cells;
- and
- (b) contacting the population of cells with a genome editing enhancer,
- wherein expression of the engineered nuclease in the presence of the genome editing enhancer increases the frequency of genome editing in the population of cells.
A method of increasing homology directed repair (HDR) in a population of cells comprising:
- (a) contacting the population of cells with a genome editing enhancer;
- (b) introducing an engineered nuclease to generate a double-strand break (DSB) at a target site;
- and
- (b) introducing a donor repair template into the population of cells;
- wherein expression of the engineered nuclease in the presence of the genome editing enhancer and the donor repair template increases the frequency of incorporation of the donor repair template at the target site by homology directed repair (HDR).
A method of increasing non-homologous end joining (NHEJ) in a population of cells comprising:
- (a) contacting the population of cells with a genome editing enhancer;
- (b) introducing an engineered nuclease to generate a double-strand break (DSB) at a target site;
- (a) introducing an engineered nuclease into a population of cells;
- and
- wherein expression of the engineered nuclease in the presence of the genome editing enhancer increases the frequency of NHEJ at the target site.
Dependencies are inferred from the claim text.