Inventors:
Stanley F. Nelson - Los Angeles CA, US
Carrie Miceli - Los Angeles CA, US
Miriana Moran - Los Angeles CA, US
Assignee:
The Regents of the University of california - Oakland CA
International Classification:
A61K 31/4178, C12Q 1/68, A61K 31/7105
US Classification:
514 44 A, 435375, 514392, 506 10, 435 613, 435 612, 506 9, 435 611
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.