Pharmaceutical composition for treatment of Duchenne muscular dystrophy
AssigneesJCR Pharmaceuticals Co., Ltd., Masafumi Matsuo
InventorsMasafumi Matsuo, Shoichiro Kamei
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Abstract
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.
Claims2
An isolated and purified oligonucleotide selected from the group consisting of:
- a DNA consisting of a nucleotide sequence set forth as SEQ ID NO:15 in the Sequence Listing, and
- an RNA consisting of a nucleotide sequence set forth as SEQ ID NO:15 in the Sequence Listing in which all thymine nucleotides are replaced by uracil.
An isolated and purified antisense oligonucleotide consisting of a nucleotide sequence fully complementary to the nucleotide sequence set forth as SEQ ID NO:15 in the Sequence Listing.
Dependencies are inferred from the claim text.