WO2001079524A3 - Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof - Google Patents

Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof Download PDF

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Publication number
WO2001079524A3
WO2001079524A3 PCT/US2001/012130 US0112130W WO0179524A3 WO 2001079524 A3 WO2001079524 A3 WO 2001079524A3 US 0112130 W US0112130 W US 0112130W WO 0179524 A3 WO0179524 A3 WO 0179524A3
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WO
WIPO (PCT)
Prior art keywords
pathogen
specific
relates
resistant
toxic agents
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/US2001/012130
Other languages
French (fr)
Other versions
WO2001079524A2 (en
Inventor
James S Norris
Gary A Clawson
Caroline Westwater
David Schofield
Michael G Schmidt
Brian Hoel
Joseph Dolan
Wei-Hua Pan
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.)
University of South Carolina
Medical University of South Carolina MUSC
Penn State Research Foundation
Original Assignee
University of South Carolina
Medical University of South Carolina MUSC
Penn State Research Foundation
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Filing date
Publication date
Application filed by University of South Carolina, Medical University of South Carolina MUSC, Penn State Research Foundation filed Critical University of South Carolina
Priority to AU5347101A priority Critical patent/AU5347101A/en
Priority to EP01926973A priority patent/EP1397489A4/en
Priority to CA002406403A priority patent/CA2406403A1/en
Priority to AU2001253471A priority patent/AU2001253471B2/en
Priority to JP2001577507A priority patent/JP2004525602A/en
Priority to US10/257,480 priority patent/US20040220123A1/en
Publication of WO2001079524A2 publication Critical patent/WO2001079524A2/en
Anticipated expiration legal-status Critical
Publication of WO2001079524A3 publication Critical patent/WO2001079524A3/en
Priority to US11/375,690 priority patent/US20060223774A1/en
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/12—Keratolytics, e.g. wart or anti-corn preparations
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00—Antiparasitic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure

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

Abstract

The present invention relates to the discovery, identification and characterization of toxic agents which are lethal to pathogens and methods for targeting such toxic agents to a pathogen or pathogen infected cells in order to treat and/or eradicate the infection. In particular, the present invention relates to toxic agents which target bacteria at different stages of the bacterial life cycle, which are delivered alone or in combination to bacteria or bacteria-infected cells. The invention relates to toxic agents which are lethal to diseased cells and methods for targeting such toxic agent to a diseased cell in order to treat and/or eradicate the disease. The present invention relates to promoter elements which are pathogen-specific or tissue-specific and the use of such promoter elements to achieve pathogen-specific or tissue-specific expression of the toxic agent(s) and/or ribozyme(s) of the present invention. Specifically, the invention relates to the delivery of one or more toxic gene products, antisense RNAs, or ribozymes, or combination thereof. The invention provides a novel system by which multiple pathogenic targets may be simultaneously targeted to cause the death of a pathogen, or cell infected with a pathogen. Further, the invention has important implications in the eradication of drug-resistant bacterium and bacterial pathogens. The invention provides a novel system by which multiple targets may be simultaneously targeted to cause the death of a diseased cell. The invention has important implications in the eradication of drug-resistant pathogens (such as antibiotic resistant bacteria) and drug-resistant diseased cells (such as drug-resistant cancer cells).
PCT/US2001/012130 1999-04-14 2001-04-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof Ceased WO2001079524A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
AU5347101A AU5347101A (en) 2000-04-13 2001-04-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof
EP01926973A EP1397489A4 (en) 2000-04-13 2001-04-13 TOXIC AGENTS, RIBOYZMS, ANDYZAMES AND ANTISENSE OLIGONUCLEOTIDES SPECIFIC TO TISSUES AND PATHOGENS AND METHOD OF USE THEREOF
CA002406403A CA2406403A1 (en) 2000-04-13 2001-04-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof
AU2001253471A AU2001253471B2 (en) 2000-04-13 2001-04-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof
JP2001577507A JP2004525602A (en) 2000-04-13 2001-04-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, DNAzymes and antisense oligonucleotides and methods of using them
US10/257,480 US20040220123A1 (en) 1999-04-14 2001-04-13 Tissue-specific and pathogens-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof
US11/375,690 US20060223774A1 (en) 2000-12-07 2006-03-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US54844900A 2000-04-13 2000-04-13
US09/548,449 2000-04-13
US25181000P 2000-12-07 2000-12-07
US60/251,810 2000-12-07

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US11/375,690 Division US20060223774A1 (en) 2000-12-07 2006-03-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof

Publications (2)

Publication Number Publication Date
WO2001079524A2 WO2001079524A2 (en) 2001-10-25
WO2001079524A3 true WO2001079524A3 (en) 2004-01-08

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PCT/US2001/012130 Ceased WO2001079524A2 (en) 1999-04-14 2001-04-13 Tissue-specific and pathogen-specific toxic agents, ribozymes, dnazymes and antisense oligonucleotides, and methods of use thereof

Country Status (5)

Country Link
EP (1) EP1397489A4 (en)
JP (1) JP2004525602A (en)
AU (2) AU2001253471B2 (en)
CA (1) CA2406403A1 (en)
WO (1) WO2001079524A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7575918B2 (en) 1999-04-14 2009-08-18 The Penn State Research Foundation Tissue-specific and pathogen-specific ribozymes

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2298869A1 (en) * 2003-06-13 2011-03-23 University Of Medicine And Dentistry Of New Jersey Recombinant protein production in the presence of mRNA interferase
EP1677834B1 (en) * 2003-10-06 2014-01-08 Gangagen, Inc. Defined dose therapeutic phage
SG11201604198YA (en) 2013-12-30 2016-07-28 Curevac Ag Methods for rna analysis
WO2019171191A1 (en) * 2018-03-05 2019-09-12 Dr. Reddy's Institute Of Life Sciences Embryonic zebrafish models using dnazyme mediated knockdown
CN118340133A (en) * 2024-04-24 2024-07-16 农业农村部环境保护科研监测所 An ecological technology method for efficiently reducing antibiotic resistance genes in cattle dung

Citations (3)

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US5989804A (en) * 1994-07-08 1999-11-23 New England Medical Center Hospitals, Inc. E6 binding proteins
US6004513A (en) * 1994-12-27 1999-12-21 Naxcor Automatic device for nucleic acid sequence detection employing amplification probes
US6015694A (en) * 1993-09-15 2000-01-18 Chiron Corporation Method for stimulating an immune response utilizing recombinant alphavirus particles

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DE69033850T2 (en) * 1989-12-01 2002-06-06 Vysis, Inc. Nucleic acid probes for the detection of HPV transcripts
GB9015845D0 (en) * 1990-07-19 1990-09-05 Emery Vincent C Diagnostic method
FR2679254B1 (en) * 1991-07-17 1993-11-19 Clonatec OLIGONUCLEOTIDE PROBES FOR THE TYPING AND DETECTION OF HUMAN PAPILLOMAVIRUSES.
EP0642589A4 (en) * 1992-05-11 1997-05-21 Ribozyme Pharm Inc Method and reagent for inhibiting viral replication.
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US5985662A (en) * 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
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US20040209263A1 (en) * 2000-12-07 2004-10-21 Clawson Gary A. Selection of catalytic nucleic acids targeted to infectious agents

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
US6015694A (en) * 1993-09-15 2000-01-18 Chiron Corporation Method for stimulating an immune response utilizing recombinant alphavirus particles
US5989804A (en) * 1994-07-08 1999-11-23 New England Medical Center Hospitals, Inc. E6 binding proteins
US6004513A (en) * 1994-12-27 1999-12-21 Naxcor Automatic device for nucleic acid sequence detection employing amplification probes

Non-Patent Citations (1)

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Title
See also references of EP1397489A4 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7575918B2 (en) 1999-04-14 2009-08-18 The Penn State Research Foundation Tissue-specific and pathogen-specific ribozymes

Also Published As

Publication number Publication date
AU5347101A (en) 2001-10-30
EP1397489A2 (en) 2004-03-17
EP1397489A4 (en) 2005-05-11
WO2001079524A2 (en) 2001-10-25
CA2406403A1 (en) 2001-10-25
AU2001253471B2 (en) 2007-07-26
JP2004525602A (en) 2004-08-26

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