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.
本发明涉及一种增强相关前mRNA中外显子跳越的方法,该方法包括使前mRNA与有效量的选自表1所示化合物的小分子或其药学上可接受的盐、
水合物、溶液或异构体接触,以及与针对前mRNA中剪接序列特异的反义寡核苷酸接触。