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Identification of small molecules that facilitate therapeutic exon skipping

  • US 10,188,633 B2
  • Filed: 03/30/2016
  • Issued: 01/29/2019
  • Est. Priority Date: 08/30/2011
  • Status: Active Grant
First Claim
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1. A method for enhancing exon skipping in an mRNA of interest, comprising contacting the mRNA with an antisense oligonucleotide that is specific for a splicing sequence in the mRNA and an effective amount of dantrolene, Ryanodine, or RyCal S107, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof.

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