Medicament for treatment of Duchenne muscular dystrophy

• US 6,653,467 B1
• Filed: 09/22/2000
• Issued: 11/25/2003
• Est. Priority Date: 04/26/2000
• Status: Expired due to Term

• Current Assignee: JCR Pharmaceuticals Co., Ltd., Masafumi Matsuo
• Original Assignee: JCR Pharmaceuticals Co., Ltd., Masafumi Matsuo
• Inventors: Takeshima, Yasuhiro, Matsuo, Masafumi
• Primary Examiner(s): LACOURCIERE, KAREN A
• Application Number:
  US09/667,327
• Time in Patent Office: 1,159 Days
• Field of Search: 435/6, 435/91.1, 435/91.3, 435/325, 435/375, 536/23.1, 536/23.2, 536/24.3, 536/24.31, 536/24.33, 536/24.5, 514/44
• US Class Current: 536/24.5
• CPC Class Codes: A61K 38/00 Medicinal preparations containing peptides peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00
• CPC Class Codes: A61K 48/00 Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
• CPC Class Codes: A61P 21/00 Drugs for disorders of the muscular or neuromuscular system
• CPC Class Codes: A61P 21/04 for myasthenia gravis
• CPC Class Codes: A61P 21/06 Anabolic agents androgens A61P5/26
• CPC Class Codes: C12N 15/113 Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing when used in plants C12N15/8218
• CPC Class Codes: C12N 2310/11 Antisense
• CPC Class Codes: C12N 2310/315 Phosphorothioates

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.

Claims

1. 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.

2. 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.