Cell based methods and systems for the identification of RNA regulatory sequences and compounds that modulate their functions
AssigneePTC Therapeutics, Inc.
InventorMatthew C. Pelligrini
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AssigneePTC Therapeutics, Inc.
InventorMatthew C. Pelligrini
Estimating the term…
The present invention is directed to methods of screening for a small molecule that modulates the ability of a RNA regulatory sequence to inhibit mRNA translation in a mammalian cell comprising incubating the mammalian cell expressing a reporter mRNA and the RNA regulatory sequence in the presence of the small molecule, wherein the RNA regulatory sequence is not attached to the reporter mRNA; and monitoring a reporter protein signal produced by the reporter mRNA, wherein a change in the signal of the reporter protein in the presence of the small molecule compared to the reporter protein signal in the absence of the small molecule indicates the small molecule modulates the ability of the RNA regulatory sequence to inhibit mRNA translation.
A method of identifying a small molecule compound that inhibits the ability of an hepatitis C virus-internal ribosome entry site (HCV-IRES) mRNA regulatory sequence transcript fragment encoded by a sequence consisting of SEQ ID NO:
A method of identifying a small molecule compound that inhibits an hepatitis C virus-internal ribosome entry site (HCV-IRES) mRNA regulatory sequence transcript fragment encoded by a sequence selected from the group consisting of—SEQ ID NO 20, SEQ ID NO 32, SEQ ID NO 38, SEQ ID NO 41, and SEQ ID NO 44 from inhibiting mRNA translation in a mammalian cell culture comprising
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