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US 10,196,639 B2Granted
Modulation of gene expression and screening for deregulated protein expression
AssigneeUniversity of Southampton
InventorsIgor Vorechovsky, Jana Kralovicova
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Abstract
Disclosed herein include compositions and methods of modulating protein expression that utilizes an activator or a repressor of a non-sense mediated RNA decay switch exon (NSE). In some embodiments, also included herein are compositions and methods of modulating protein expression that uses an agent that targets a transposed element.
Claims20
A method of modulating protein expression, comprising:
- a) contacting a non-sense mediated RNA decay switch exon (NSE)-repressor agent to a target motif within a pre-processed mRNA transcript, wherein the target motif is located in an intronic region between two canonical exons;
- b) processing a mRNA transcript from the pre-processed mRNA transcript thereby forming a processed mRNA transcript, wherein the NSE-repressor agent modulates exclusion of an NSE from the processed mRNA transcript;
- and
- c) translating the processed mRNA transcript wherein the exclusion of the NSE upregulates protein expression relative to the protein expression of an equivalent mRNA transcript comprising the NSE.
1 more element
A method of treating or preventing a disease or condition in a subject in need thereof, the method comprising:
- administering to the subject a pharmaceutical composition comprising:
- (i) a non-sense mediated RNA decay switch exon (NSE)-repressor agent that interacts with a target motif within a pre-processed mRNA transcript to modulate exclusion of an NSE from a processed mRNA transcript wherein the target motif is located in an intronic region between two canonical exons:
- 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 NSE-repressor agent by an increase in expression of a protein translated from the processed mRNA transcript.
2 more elements
Dependencies are inferred from the claim text.