Reducing intron retention
AssigneeUniversity of Southampton
InventorsIgor Vorechovsky, Jana Kralovicova
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Abstract
Disclosed herein are methods, compositions, polynucleic acid polymers, assays, and kits for inducing processing of a partially processed mRNA transcript to remove a retained intron to produce a fully processed mRNA transcript that encodes a full-length functional form of a protein. Also described herein are methods and compositions for treating a disease or condition characterized by impaired production of a full-length functional form of a protein or for treating a disease or condition characterized by a defective splicing in a subject.
Claims24
A method of treating or preventing a disease or condition characterized by an impaired production of a functional protein or by a defective splicing in a subject comprising correction of intron retention in gene transcripts, the method comprising:
- administering to the subject a pharmaceutical composition comprising:
- (i) a therapeutic agent that induces splicing out of an entire intron in a partially processed mRNA transcript, wherein the therapeutic agent comprises a polynucleic acid polymer or a small molecule;
- and
- (ii) a pharmaceutically acceptable excipient and/or a delivery vehicle;
- wherein the disease or condition characterized by an impaired production of a functional protein or by a defective splicing is treated or prevented in the subject by the administration of the therapeutic agent.
Dependencies are inferred from the claim text.