WO1997048795A3 - Compositions and methods for treating specific gene expression-related diseases and disorders in humans - Google Patents

Compositions and methods for treating specific gene expression-related diseases and disorders in humans Download PDF

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Publication number
WO1997048795A3
WO1997048795A3 PCT/US1997/010143 US9710143W WO9748795A3 WO 1997048795 A3 WO1997048795 A3 WO 1997048795A3 US 9710143 W US9710143 W US 9710143W WO 9748795 A3 WO9748795 A3 WO 9748795A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
disorders
compositions
humans
gene expression
Prior art date
Application number
PCT/US1997/010143
Other languages
French (fr)
Other versions
WO1997048795A2 (en
Inventor
Paul J Schechter
Russell R Martin
Christophe Tournerie
Sudhir Agrawal
Robert W Coombs
Original Assignee
Hybridon Inc
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 Hybridon Inc filed Critical Hybridon Inc
Priority to AU33096/97A priority Critical patent/AU3309697A/en
Publication of WO1997048795A2 publication Critical patent/WO1997048795A2/en
Publication of WO1997048795A3 publication Critical patent/WO1997048795A3/en

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Classifications

    • 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
    • C12N15/1131—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 viruses
    • C12N15/1132—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 viruses against retroviridae, e.g. HIV
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • AIDS & HIV (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention provides therapeutic compositions and methods for treating humans suffering from diseases or disorders caused by cellular expression of aberrant exogenous genes or aberrant endogenous genes comprising administering to the human a therapeutically effective amount of a composition of the invention comprising an oligonucleotide capable of specifically down-regulating the expression of such a gene. A specific example is given for the treatment of HIV infections.
PCT/US1997/010143 1996-06-18 1997-06-11 Compositions and methods for treating specific gene expression-related diseases and disorders in humans WO1997048795A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU33096/97A AU3309697A (en) 1996-06-18 1997-06-11 Compositions and methods for treating specific gene expression-related diseases and disorders in humans

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US2041796P 1996-06-18 1996-06-18
US60/020,417 1996-06-18

Publications (2)

Publication Number Publication Date
WO1997048795A2 WO1997048795A2 (en) 1997-12-24
WO1997048795A3 true WO1997048795A3 (en) 1998-04-30

Family

ID=21798518

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1997/010143 WO1997048795A2 (en) 1996-06-18 1997-06-11 Compositions and methods for treating specific gene expression-related diseases and disorders in humans

Country Status (2)

Country Link
AU (1) AU3309697A (en)
WO (1) WO1997048795A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19855469C2 (en) * 1998-12-01 2001-01-11 Michael Esrich Method for determining the apolipoprotein E genotype in a human sample
US8785409B2 (en) 2007-01-30 2014-07-22 Geron Corporation Compounds having anti-adhesive effects on cancer cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992003456A1 (en) * 1990-08-16 1992-03-05 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating the effects of cytomegalovirus infections
US5442049A (en) * 1992-11-19 1995-08-15 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating the effects of cytomegalovirus infections
WO1995032213A1 (en) * 1994-05-25 1995-11-30 Hybridon, Inc. Oligonucleotides with anti-cytomegalovirus activity

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992003456A1 (en) * 1990-08-16 1992-03-05 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating the effects of cytomegalovirus infections
US5442049A (en) * 1992-11-19 1995-08-15 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating the effects of cytomegalovirus infections
WO1995032213A1 (en) * 1994-05-25 1995-11-30 Hybridon, Inc. Oligonucleotides with anti-cytomegalovirus activity

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
COHEN, J.: "AIDS therapy. New hope against blindness [news].", SCIENCE, (1995 APR 21) 268 (5209) 368-9., XP002046611 *
CROOKE S T: "PROGRESS IN ANTISENSE THERAPEUTICS", HEMATOLOGIC PATHOLOGY, vol. 9, no. 2, 1995, pages 59 - 72, XP000651301 *
PUTNAM D A: "ANTISENSE STRATEGIES AND THERAPEUTIC APPLICATIONS", AMERICAN JOURNAL OF HEALTH-SYSTEM PHARMACY, vol. 53, no. 2, 15 January 1996 (1996-01-15), pages 151 - 160, 182/183, XP000603121 *

Also Published As

Publication number Publication date
WO1997048795A2 (en) 1997-12-24
AU3309697A (en) 1998-01-07

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