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US 2026/0308550 A1Published application
Novel type vi crispr orthologs and systems
AssigneesThe Broad Institute, Inc., Massachusetts Institute of Technology
InventorsFeng Zhang, David Benjamin Turitz Cox
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Abstract
The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.
Claims28
A method of modifying a target RNA sequence, said method comprising delivering to said target RNA sequence a composition comprising:
- (a) a Cas13b effector protein optimized for activity in a mammalian cell,
- (b) a guide molecule capable of forming a complex with the Cas13b and directing sequence-specific binding of the complex to a target sequence of a target RNA, wherein the target RNA sequence comprises one or more genes associated with disease, and
- (c) one or more heterologous functional domains fused to the Cas13b effector protein,
- wherein the one or more heterologous functional domains modifies the target RNA sequence,
- wherein the Cas13b effector protein is at least 99% homologous to the Cas13b effector protein selected from the group consisting of SEQ ID NO:
- 146-156, 158-163, and 165-166.
3 more elements
Dependencies are inferred from the claim text.