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IDENTIFICATION OF STRUCTURALLY SIMILAR SMALL MOLECULES THAT ENHANCE THERAPEUTIC EXON SKIPPING
IDENTIFICATION OF STRUCTURALLY SIMILAR SMALL MOLECULES THAT ENHANCE THERAPEUTIC EXON SKIPPING
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机译:结构相似的小分子的识别,可增强治疗性外显子跳跃
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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 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.
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