Pharmaceutical composition for treatment of Duchenne muscular dystrophy

• US 6,727,355 B2
• Filed: 08/16/2001
• Issued: 04/27/2004
• Est. Priority Date: 08/25/2000
• Status: Expired due to Term

• Current Assignee: JCR Pharmaceuticals Co., Ltd., Masafumi Matsuo
• Original Assignee: JCR Pharmaceuticals Co., Ltd., Masafumi Matsuo
• Inventors: Matsuo, Masafumi, Kamei, Shoichiro
• Primary Examiner(s): LACOURCIERE, KAREN A
• Application Number:
  US09/930,251
• Publication Number:
  US 20020055481A1
• Time in Patent Office: 985 Days
• Field of Search: 435/6, 435/91.1, 435/91.8, 435/325, 435/375, 536/23.1, 536/23.2, 536/24.5, 536/24.3, 536/24.31, 536/24.33, 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 47/02 Inorganic compounds
• CPC Class Codes: A61K 47/26 Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
• CPC Class Codes: A61K 9/0019 Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner non-active ingredients are additionally classified in A61K47/00
• CPC Class Codes: A61P 21/00 Drugs for disorders of the muscular or neuromuscular system
• CPC Class Codes: C07K 14/4708 Duchenne dystrophy

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.

Claims

1. 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.

2. 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.