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.
Claims17
A method of making a polynucleic acid for inducing processing of a partially processed mRNA transcript to facilitate removal of a retained intron to produce a fully processed mRNA transcript that encodes a functional form of a protein, the method comprising:
- synthesizing the polynucleic acid, wherein the polynucleic acid is from about 10 to about 50 nucleotides in length and comprises a sequence that is complementary to at least 10 contiguous bases of a wild-type target sequence of the partially processed mRNA transcript, wherein the partially processed mRNA transcript is capable of encoding the functional form of a protein and comprises at least one retained intron, wherein the polynucleic acid induces splicing out of the retained intron from the partially processed mRNA transcript.
Dependencies are inferred from the claim text.