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US 12,559,742 B2Granted
Donor repair templates multiplex genome editing
AssigneeNovo Nordisk A/S
InventorsBaeckseung Lee, Alexander Astrakhan
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Abstract
The present disclosure provides improved multiplex genome editing compositions and methods. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.
Claims20
A method of editing the genome of a cell at multiple target sites by introducing one or more engineered nucleases and a DNA donor repair template, wherein the DNA donor repair template comprises from 5′ to 3′:
- (a) a 5′homology arm homologous to a DNA sequence 5′ of a first target site;
- (b) a 5′homology arm homologous to a DNA sequence 5′ of a second target site;
- (c) a transgene;
- (d) a 3 ‘homology arm homologous to a DNA sequence 3’ of the second target site;
- and
- (e) a 3′homology arm homologous to a DNA sequence 3′ of the first target site.
3 more elements
Dependencies are inferred from the claim text.