Transcription modulation in animals using crispr/cas systems
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsCharleen Hunt, Suzanne Hartford, Guochun Gong, Brian Zambrowicz
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Abstract
Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.
Claims26
-109. (canceled)
A rodent comprising a first genomically integrated expression cassette, wherein the first expression cassette comprises:
- (a) a nucleic acid encoding a chimeric Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) associated (Cas) protein comprising a nuclease-inactive Cas protein fused to one or more transcriptional activation domains;
- and
- (b) a nucleic acid encoding a chimeric adaptor protein comprising an adaptor protein fused to one or more transcriptional activation domains, wherein the adaptor protein is a protein that specifically recognizes and binds to a distinct RNA sequence;
- wherein the first expression cassette is integrated into a Rosa26 locus, and
- wherein the first expression cassette is operably linked to an endogenous Rosa26 promoter of the Rosa26 locus.
A rodent cell comprising a genomically integrated expression cassette, wherein the expression cassette comprises:
- (a) a nucleic acid encoding a chimeric Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) associated (Cas) protein comprising a nuclease-inactive Cas protein fused to one or more transcriptional activation domains;
- and
- (b) a nucleic acid encoding a chimeric adaptor protein comprising an adaptor protein fused to one or more transcriptional activation domains, wherein the adaptor protein is a protein that specifically recognizes and binds to a distinct RNA sequence;
- wherein the expression cassette is integrated into a Rosa26 locus, and
- wherein the expression cassette is operably linked to an endogenous Rosa26 promoter of the Rosa26 locus.
Dependencies are inferred from the claim text.