Reducing intron retention
AssigneeUniversity of Southampton
InventorsIgor Vorechovsky, Jana Kralovicova
Life of this patent
Estimating the term…
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 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;
- and
- (ii) a pharmaceutically acceptable excipient and/or a delivery vehicle;
- wherein the disease or condition is treated or prevented in the subject by the administration of the therapeutic agent.
(canceled)
(canceled)
(canceled)
-30. (canceled)
Dependencies are inferred from the claim text.