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IDENTIFICATION OF SMALL MOLECULES THAT FACILITATE THERAPEUTIC EXON SKIPPING

  • US 20140080896A1
  • Filed: 09/13/2013
  • Published: 03/20/2014
  • Est. Priority Date: 08/30/2011
  • Status: Abandoned Application
First Claim
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1. A method for enhancing exon skipping in an mRNA of interest, comprising contacting the mRNA with an effective amount of a small molecule compound selected from one or more of furaltadone hydrochloride, 5-iodotubericidin, bendroflumethiazide, cyclopiazonic acid, GW 5074, indirubin, rescinnamin, U-0126, acetopromazine maleate salt, Ro 31-8220, dantrolene, dichlorobenzamil, ellipticine, fenbendazole, GF 109203×

  • , halofantrine, niclosamide, pimozide, reserpine, syringospine, a RyCal, piperacetazine, fluphenazine dihydrochloride, trifluorperazine dihydrochloride, yohimbinic acid, or menadione,or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof.

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