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WO1993001286A3 - Localized oligonucleotide therapy - Google Patents

Localized oligonucleotide therapy Download PDF

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Publication number
WO1993001286A3
WO1993001286A3 PCT/US1992/005305 US9205305W WO9301286A3 WO 1993001286 A3 WO1993001286 A3 WO 1993001286A3 US 9205305 W US9205305 W US 9205305W WO 9301286 A3 WO9301286 A3 WO 9301286A3
Authority
WO
WIPO (PCT)
Prior art keywords
localized
antisense oligonucleotides
gene
oligonucleotide therapy
application
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US1992/005305
Other languages
French (fr)
Other versions
WO1993001286A2 (en
Inventor
Robert D Rosenberg
Michael Simons
Elazer Edelman
Robert S Langer
Jean-Luc Dekeyser
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Massachusetts Institute of Technology
Original Assignee
Massachusetts Institute of Technology
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Massachusetts Institute of Technology filed Critical Massachusetts Institute of Technology
Priority to JP5502240A priority Critical patent/JPH07501204A/en
Priority to AU23032/92A priority patent/AU659482B2/en
Publication of WO1993001286A2 publication Critical patent/WO1993001286A2/en
Publication of WO1993001286A3 publication Critical patent/WO1993001286A3/en
Priority to NO934828A priority patent/NO934828L/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Dermatology (AREA)
  • Epidemiology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Disclosed is a method for localized application of antisense oligonucleotides, which has been found to be effective in inhibiting expression and translation of a variety of genes. The method utilizes antisense oligonucleotides which are specific for the mRNA transcribed from the gene of interest. The antisense oligonucleotides are applied directly to the desired locus of the cells to be treated, where they hybridize with the mRNA and inhibit expression of the gene. Devices for localized antisense application and methods for making them also are described.
PCT/US1992/005305 1991-06-28 1992-06-23 Localized oligonucleotide therapy Ceased WO1993001286A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP5502240A JPH07501204A (en) 1991-06-28 1992-06-23 Topical oligonucleotide therapy
AU23032/92A AU659482B2 (en) 1991-06-28 1992-06-23 Localized oligonucleotide therapy
NO934828A NO934828L (en) 1991-06-28 1993-12-27 Localized oligonucleotide therapy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US72345491A 1991-06-28 1991-06-28
US723,454 1991-06-28

Publications (2)

Publication Number Publication Date
WO1993001286A2 WO1993001286A2 (en) 1993-01-21
WO1993001286A3 true WO1993001286A3 (en) 1993-04-01

Family

ID=24906337

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1992/005305 Ceased WO1993001286A2 (en) 1991-06-28 1992-06-23 Localized oligonucleotide therapy

Country Status (3)

Country Link
JP (1) JPH07501204A (en)
AU (1) AU659482B2 (en)
WO (1) WO1993001286A2 (en)

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US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA
WO2024074668A1 (en) 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
WO2024074670A1 (en) 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
WO2024105063A1 (en) 2022-11-15 2024-05-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 39-40
WO2025036833A1 (en) 2023-08-11 2025-02-20 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 53

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JOURNAL OF BIOLOGICAL CHEMISTRY vol. 264, no. 12, 25 April 1989, BALTIMORE, MD US pages 6990 - 6995 REILLY, C.F. ET AL. 'Heparin prevents vascular smooth muscle cell progression through the G1 phase of the cell cycle' cited in the application *
JOURNAL OF BIOLOGICAL CHEMISTRY vol. 267, no. 7, 5 March 1992, BALTIMORE, MD US pages 4625 - 4630 BROWN, K.E. ET AL. 'Expression of the c-myb proto-oncogene in bovine vascular smooth muscle cells' *
JOURNAL OF THE NATIONAL CANCER INSTITUTE vol. 81, no. 20, 18 October 1989, pages 1539 - 1545 ROTHENBERG, M. ET AL. 'Oligodeoxynucleotides as anti-sense inhibitors of gene expression: therapeutic implications' *
NUCLEIC ACIDS RESEARCH vol. 19, no. 4, 25 February 1991, ARLINGTON, VIRGINIA US pages 747 - 750 SHAW, J.-P. ET AL. 'Modified deoxyoligonucleotides stable to exonuclease degradation in serum' cited in the application *
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA vol. 86, May 1989, WASHINGTON US pages 3379 - 3383 ANFOSSI, G. ET AL. 'An oligomer complementary to c-myb-encoded mRNA inhibits proliferation of human myeloid leukemia cell lines' cited in the application *

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US7255874B1 (en) 2001-12-21 2007-08-14 Closure Medical Corporation Biocompatible polymers and adhesives: compositions, methods of making and uses related thereto
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA
WO2024074668A1 (en) 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
WO2024074670A1 (en) 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
WO2024105063A1 (en) 2022-11-15 2024-05-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 39-40
WO2025036833A1 (en) 2023-08-11 2025-02-20 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 53

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JPH07501204A (en) 1995-02-09

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