Reducing intron retention
First Claim
1. 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.
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Accused Products
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.
237 Citations
24 Claims
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1. 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:
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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. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24)
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Specification