Pharmaceutical Composition For Treatment Of Duchenne Muscular Dystrophy


The invention provides an isolated and purified DNA set forth as SEQ ID NO:15 in the Sequence Listing and an antisense oligonucleotide complementary to the DNA. The DNA represents the splicing enhancer sequence (SES) in exon 45 of human dystrophin gene, and serves as a template in preparation of the antisense oligonucleotide, which is used to induce exon 45 skipping in certain group of patient with Duchenne muscular dystrophy to restore the reading frame of dystrophin mRNA.

Download PDF
Document Preview
Document History
  • Publication: Apr 27, 2004
  • Application: Aug 16, 2001
    US US 93025101 A
  • Priority: Aug 25, 2000
    JP 2000256547 A

Download Citation

Sign in to the Lens