Nonsense-mediated mrna decay
OwnerLynne Maquatinventor
Life of this patent
Estimating the term…
Abstract
Disclosed are compositions and methods relating to nonsense mediated decay (NMD) particularly in the “pioneer” round of translation. The present invention provides methods of treating disorders relating to NMD and methods of screening substances for use in the study of or treatment of conditions relating to NMD.
Claims73
A method of treating a disorder in a subject comprising administering to the subject a substance, wherein the substance modulates of nonsense-mediated mRNA decay in the pioneer round of translation.
A method of screening for a substance that modulates a nonsense-mediated mRNA decay (NMD) complex comprising
- (a) incubating the substance with the complex, and
- (b) assaying for a change in NMD, an increase or decrease in NMD activity indicating a modulating substance.
A method of screening for a substance that modulates nonsense-mediated mRNA decay (NMD) comprising
- (a) incubating the substance with a stably transfected cell comprising a reporter gene with a nonsense-mutation, and
- (b) assaying the amount of NMD in the cell, wherein a increase or decrease in the amount of mRNA relative to the amount of mRNA in the absence of the substance indicates a substance that modulates NMD activity.
A method of screening for a substance that inhibits nonsense-mediated mRNA decay (NMD) comprising
- (a) incubating the substance with Upf2 and Upf3 forming a substance-Upf2-Upf3 mixture, and
- (b) assaying the amount of Upf2-Upf3 complex present in the mixture, wherein a decrease in the amount of Upf2-Upf3 complex relative to the amount of Upf2-Upf3 complex in the absence of the substance indicates that the substance inhibits NMD.
A method of screening for a substance that inhibits nonsense-mediated mRNA decay (NMD) comprising
- (a) incubating the substance with Upf2 and Upf3X forming a substance-Upf2-Upf3X mixture, and
- (b) assaying the amount of Upf2-Upf3X complex present in the mixture, wherein a decrease in the amount of Upf2-Upf3X complex relative to the amount of Upf2-Upf3X complex in the absence of the substance indicates that the substance inhibits NMD.
A method of screening for a substance that modulates nonsense-mediated mRNA decay (NMD) comprising,
- a) administering a substance to a system, wherein the system comprises the components essential for NMD activity, and
- b) assaying the effect of the substance on the amount of NMD activity in the system, wherein a substance which causes a change in the amount of NMD activity present in the system compared to the amount of NMD activity in the system in the absence of composition is a modulator.
A method of making a modulator of nonsense-mediated mRNA decay (NMD) comprising,
- a) administering a substance to a system, wherein the system comprises NMD activity,
- b) assaying the effect of the substance on the amount of NMD activity in the system,
- c) selecting a substance which causes a change in the amount of NMD activity present in the system compared to the amount of NMD activity in the system in the absence of substance, and
- d) synthesizing the substance.
A substance that modulates NMD in the pioneer round of translation.
A vector comprising a nucleic acid that encodes a protein that modulates NMD.
Dependencies are inferred from the claim text.