Programmable rna scaffolds for multivariate effector recruitment using crispr/cas
AssigneeWilliam Marsh Rice University
InventorsIsaac HILTON, Barun MAHATA, Alan CABRERA
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Abstract
The present disclosure is directed to CRISPR systems comprising an RNA scaffold that can recruit up to four different effector domains to human loci. The RNA scaffolds may comprise at least three different types of stem loops that each specifically bind to a different stem-loop binding protein. The stem-loop binding proteins may then be fused to the different effector domains, one of which is a Cas9 protein, to bring all of the components together at one site.
Claims20
A composition comprising one or more nucleic acid(s) encoding (i) a first effector protein fused to a first stem-loop binding protein, wherein the first effector protein is a CRISPR/Cas protein, (ii) a second effector protein fused with a second stem-loop binding protein, (iii) an RNA molecule comprising at least one of a first stem loop that binds to the first stem-loop binding protein and at least one of a second stem loop that binds to the second stem-loop binding protein, and (iv) a guide RNA(s) (gRNA) targeting at least one genomic locus.
A viral vector comprising nucleic acid sequences encoding (ii) a second effector protein fused with a second stem-loop binding protein, (iii) an RNA molecule comprising at least one of a first stem loop that binds to the first stem-loop binding protein and at least one of a second stem loop that binds to the second stem-loop binding protein, and (iv) a guide RNA(s) (gRNA) targeting at least one genomic locus.
An engineered stem loop that specifically binds to CsrA, wherein the engineered stem loop is CsrB-SL Type 1 or CsrB-SL Type 2.
Dependencies are inferred from the claim text.