Improved gene editing
AssigneeOslo Universitetssykehus Hf
InventorsAdam Brian Robertson, Julia Robertson, Anika Reifschneider, Arne Klungland
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Abstract
The present invention generally relates to systems, methods and compositions used for the control of gene expression involving sequence targeting, such as genome perturbation or gene-editing, that may use vector systems related to recombinases and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and components thereof.
Claims27
A method of altering expression of at least one gene product and/or genome editing comprising:
- introducing into a cell a) an enzyme that introduces a double stranded break in a specific targeted sequence in a gene encoding said gene product in said genome of said cell, b) a nucleic acid molecule encoding a nucleic acid sequence of interest to be inserted into said genome at said double stranded break by homologous recombination;
- and c) a recombinase;
- wherein components (a), (b) and (c) are expressed in said cell so that a targeted double stranded break is introduced into the genome of said host cell and said nucleic acid molecule encoding a nucleic acid sequence of interest is inserted by homologous recombination at said double stranded break to effect altered expression of at said at least one gene product and/or genome editing of said gene encoding said gene product of interest and wherein said enzyme that introduces a double stranded break in a specific targeted sequence in a gene encoding said gene product and said nucleic acid molecule encoding a nucleic acid sequence of interest to be inserted into said genome at said double stranded break do not naturally occur together.
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An engineered, non-naturally occurring system for altering expression of a gene product and/or genome editing comprising:
- one or more nucleic acid sequences comprising a) a nucleic acid molecule encoding an enzyme that introduces a double stranded break in a specific targeted sequence in a gene encoding said gene product in said genome of said cell, b) a nucleic acid molecule encoding a nucleic acid sequence of interest to be inserted into said genome at said double stranded break;
- and c) a nucleic acid sequence encoding a recombinase;
- wherein components (a), (b) and (c) are expressed in a cell so that a targeted double stranded break is introduced into the genome of said cell and said nucleic acid molecule encoding a nucleic acid sequence of interest is inserted by homologous recombination at said double stranded break to effect altered expression of at said at least one gene product and/or genome editing of said gene encoding said gene product of interest and wherein said enzyme that introduces a double stranded break in a specific targeted sequence in a gene encoding said gene product and said nucleic acid molecule encoding a nucleic acid sequence of interest to be inserted into said genome at said double stranded break do not naturally occur together.
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Dependencies are inferred from the claim text.