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US 2025/0051764 A1Published application
Rna editing via recruitment of spliceosome components
AssigneeTacit Therapeutics, Inc.
InventorDavid A. NELLES
Life of this patent
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Abstract
Disclosed are methods and compositions for promoting trans-splicing. In some embodiments, the composition comprises an engineered small nuclear RNA that promotes trans-splicing of a target RNA molecule. The composition may further comprise an RNA donor molecule.
Claims27
A method of delivering an engineered exonic sequence to a cell, comprising:
- a. bringing the cell in contact with a composition comprising (i) a nucleic acid sequence encoding the engineered exonic sequence and (ii) a nucleic acid sequence encoding an engineered small nuclear RNA (esnRNA), wherein upon the composition coming in contact with the cell, the nucleic acid sequence encoding the engineered exonic sequence and the nucleic acid sequence encoding the esnRNA enter the cell;
- b. generating the esnRNA from the nucleic acid encoding the esnRNA;
- and
- c. using the esnRNA to promote a trans-splicing of the engineered exonic sequence to a target RNA molecule in the cell.
1 more element
Dependencies are inferred from the claim text.