Medicament for treatment of Duchenne muscular dystrophy
AssigneesJCR PHARMACEUTICAL CO., Ltd., Masafumi Matsuo
InventorsMasafumi Matsuo, Yasuhiro Takeshima
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Abstract
Antisense oligonucleotides comprising a nucleotide sequence complementary to the nucleotide sequence of SEQ ID NO:1 or NO:2 are disclosed. The antisense oligonucleotides are used for treatment of specific types of Duchenne muscular dystrophy which is attributed to a change in number of the nucleotides composing one or more exons adjacent to exon 43 or 53, respectively, in human dystrophin mRNA, wherein the change is due to deletion of one or more nucleotides from the normal nucleotide sequence for the exons, wherein the net of the change in number of the nucleotides is expressed as a reduction of (3×N+1) nucleotides, wherein N is zero or a natural number.
Claims2
An oligonucleotide selected from:
- an RNA consisting of the nucleotide sequence set forth as SEQ ID NO:
- 1, or
- a DNA consisting of the nucleotide sequence set forth as SEQ ID NO:
- 1 modified such that each uracil nucleotide of SEQ ID NO:
- 1 is replaced with thymine.
An oligonucleotide selected from:
- an RNA consisting of the nucleotide sequence set forth as SEQ ID NO:
- 2, or
- a DNA consisting of the nucleotide sequence set forth as SEQ ID NO:
- 2 modified such that each uracil nucleotide of SEQ ID NO:
- 2 is replaced with thymine.
Dependencies are inferred from the claim text.