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.

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.

Abstract: Cas9 polypeptides which target RNA and methods of using them are provided.

Abstract: Disclosed are compositions comprising: a trans-splicing nucleic acid comprising (a) one or more replacement domains that encode a therapeutic sequence; and (b) one or more intronic domains that promote RNA splicing of the replacement domain primarily in a specific tissue or cell type; and (c) one or more antisense domains that promote binding to a target RNA molecule, wherein an RNA trans-splicing reaction promotes insertion of the replacement domain into a target RNA. Methods of making and methods of using compositions of the disclosure are also provided, including but not limited to compositions of the disclosure that may be used in the treatment of a disease or disorder in a patient or subject. Example disease or disorders of the disclosure include genetic and epigenetic diseases or disorders.

Abstract: Cas9 polypeptides which target RNA and method of using them are provided.

Abstract: Provided herein are methods of identifying a cell uptake modulator of a molecule that include (a) contacting a plurality of cells of a cell-containing biological sample with a plurality of gene-editing agents, wherein a gene-editing agent from the plurality of gene-editing agents recognizes and alters a target gene of at least one cell of the plurality of cells; (b) contacting the plurality of cells with a plurality of molecules, wherein at least one molecule of the plurality of molecules is transported into at least one cell of the plurality of cells; and (c) detecting a presence of the at least one molecule in the plurality of cells, thereby identifying the cell uptake modulator of the molecule.

Abstract: Disclosed are compositions comprising: a sequence encoding a fusion protein, the sequence comprising a sequence encoding a first RNA-binding polypeptide and a sequence encoding a second RNA-binding polypeptide, wherein neither the first RNA-binding polypeptide nor the second RNA-binding polypeptide comprises a significant DNA-nuclease activity, wherein the first RNA-binding polypeptide and the second RNA-binding polypeptide are not identical, and wherein the second RNA-binding polypeptide comprises an RNA-nuclease activity. Methods of making and methods of using compositions of the disclosure are also provided. For example, compositions of the disclosure may be used in the treatment of a disease or disorder in a subject. Exemplary disease or disorders of the disclosure include genetic and epigenetic diseases or disorders.

Abstract: Cas9 polypeptides which target RNA and methods of using them are provided

Abstract: Cas9 polypeptides which target RNA and methods of using them are provided.

Abstract: Cas9 polypeptides which target RNA and methods of using them are provided.