REDUCING INTRON RETENTION

摘要

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.

主权项

1. 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.