IDENTIFICATION OF STRUCTURALLY SIMILAR SMALL MOLECULES THAT ENHANCE THERAPEUTIC EXON SKIPPING
申请人:The Regents of the University of California
公开号:US20160257952A1
公开(公告)日:2016-09-08
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 compound such as, for example, Perphenazine, Flupentixol DiHCl, Zuclopenthixol or Corynanthine HCl, or a compound which shares a similar 2-D structure and activity level with one of these compounds, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer of the compound, 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.
本发明涉及一种增强感兴趣的前mRNA的外显子跳跃的方法,包括将前mRNA与有效量的化合物接触,例如Perphenazine、Flupentixol DiHCl、Zuclopenthixol或Corynanthine HCl,或具有类似2-D结构和活性水平的化合物,或化合物的药学上可接受的盐,水合物,溶剂化物或异构体,以及选择性针对前mRNA中剪接序列的反义寡核苷酸(如果需要)。还公开了治疗杜氏肌肉萎缩症(DMD)的方法。