IDENTIFICATION OF SMALL MOLECULES THAT FACILITATE THERAPEUTIC EXON SKIPPING

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专利权人:

Stanley F. Nelson

发明人:

Stanley F. Nelson,Carrie Miceli,Miriana Moran

申请号：

US14026699

公开号：

US20140080896A1

申请日:

2013.09.13

申请国别(地区):

US

年份:

2014

代理人:

摘要：

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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