Identification of small molecules that facilitate therapeutic exon skipping
First Claim
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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Abstract
This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a small molecule selected from the compounds shown in Table 1, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.
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16 Claims
- 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.
Specification