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WO2000003004A3 - Presenilin 2 specific ribozyme - Google Patents

Presenilin 2 specific ribozyme Download PDF

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Publication number
WO2000003004A3
WO2000003004A3 PCT/EP1999/004804 EP9904804W WO0003004A3 WO 2000003004 A3 WO2000003004 A3 WO 2000003004A3 EP 9904804 W EP9904804 W EP 9904804W WO 0003004 A3 WO0003004 A3 WO 0003004A3
Authority
WO
WIPO (PCT)
Prior art keywords
presenilin
ribozyme
ribozymes
recombinant vector
alzheimer
Prior art date
Application number
PCT/EP1999/004804
Other languages
French (fr)
Other versions
WO2000003004A2 (en
Inventor
Katja Fechteler
Klaus Mendla
Nicole Sauer
Original Assignee
Boehringer Ingelheim Pharma
Katja Fechteler
Klaus Mendla
Nicole Sauer
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 Boehringer Ingelheim Pharma, Katja Fechteler, Klaus Mendla, Nicole Sauer filed Critical Boehringer Ingelheim Pharma
Priority to CA002332497A priority Critical patent/CA2332497A1/en
Priority to JP2000559226A priority patent/JP2002520016A/en
Priority to EP99934633A priority patent/EP1095138A2/en
Priority to AU50339/99A priority patent/AU5033999A/en
Publication of WO2000003004A2 publication Critical patent/WO2000003004A2/en
Publication of WO2000003004A3 publication Critical patent/WO2000003004A3/en

Links

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead

Landscapes

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

Abstract

The present invention relates to substances capable of inhibiting presenilin 2 expression in neurodegenerative diseases and in Alzheimer's disease. Ribozymes capable of cleaving presenilin 2-specific RNA are disclosed. Preferably, the invention concerns fusion ribozymes comprising a presenilin 2-specific ribozyme and an autocatalytical hammerhead ribozyme. Furthermore, recombinant DNA molecules coding for said ribozymes, a recombinant vector comprising the cDNA corresponding to said ribozymes and a host cell comprising said recombinant vector are disclosed. Additionally, the invention pertains to pharmaceutical compositions comprising said substance or ribozyme or recombinant vector and a pharmaceutically acceptable carrier. The invention also comprises the use of said substance or ribozyme or recombinant vector for the treatment of neurodegenerative diseases or for the treatment of Alzheimer's disease.
PCT/EP1999/004804 1998-07-09 1999-07-08 Presenilin 2 specific ribozyme WO2000003004A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002332497A CA2332497A1 (en) 1998-07-09 1999-07-08 Presenilin 2 specific ribozyme
JP2000559226A JP2002520016A (en) 1998-07-09 1999-07-08 Presenilin 2-specific ribozymes
EP99934633A EP1095138A2 (en) 1998-07-09 1999-07-08 Presenilin 2 specific ribozyme
AU50339/99A AU5033999A (en) 1998-07-09 1999-07-08 Presenilin 2 specific ribozyme

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP98112653.5 1998-07-09
EP98112653 1998-07-09
US12620099P 1999-03-25 1999-03-25
US60/126,200 1999-03-25

Publications (2)

Publication Number Publication Date
WO2000003004A2 WO2000003004A2 (en) 2000-01-20
WO2000003004A3 true WO2000003004A3 (en) 2000-04-20

Family

ID=45569968

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP1999/004804 WO2000003004A2 (en) 1998-07-09 1999-07-08 Presenilin 2 specific ribozyme

Country Status (6)

Country Link
EP (1) EP1095138A2 (en)
JP (1) JP2002520016A (en)
AR (1) AR020329A1 (en)
AU (1) AU5033999A (en)
CA (1) CA2332497A1 (en)
WO (1) WO2000003004A2 (en)

Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7041510B2 (en) 1996-04-25 2006-05-09 Bioarray Solutions Ltd. System and method for programmable illumination pattern generation
US7057704B2 (en) 2000-09-17 2006-06-06 Bioarray Solutions Ltd. System and method for programmable illumination pattern generation
US7056746B2 (en) 1996-04-25 2006-06-06 Bioarray Solutions Ltd. Array cytometry
US7144119B2 (en) 1996-04-25 2006-12-05 Bioarray Solutions Ltd. System and method for programmable illumination pattern generation
US7291504B2 (en) 1996-04-25 2007-11-06 Bioarray Solutions Ltd. Assay involving detection and identification with encoded particles
US7427512B2 (en) 1996-04-25 2008-09-23 Bioarray Solutions Ltd. Light-controlled electrokinetic assembly of particles near surfaces
US8691754B2 (en) 2003-09-22 2014-04-08 Bioarray Solutions, Ltd. Microparticles with enhanced covalent binding capacity and their uses
US9147037B2 (en) 2004-08-02 2015-09-29 Bioarray Solutions, Ltd. Automated analysis of multiplexed probe-target interaction patterns: pattern matching and allele identification
US9251583B2 (en) 2002-11-15 2016-02-02 Bioarray Solutions, Ltd. Analysis, secure access to, and transmission of array images
US9377388B2 (en) 1997-03-14 2016-06-28 Trustees Of Tufts College Methods for detecting target analytes and enzymatic reactions
US9436088B2 (en) 2001-06-21 2016-09-06 Bioarray Solutions, Ltd. Un-supported polymeric film with embedded microbeads

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030027126A1 (en) 1997-03-14 2003-02-06 Walt David R. Methods for detecting target analytes and enzymatic reactions
US9709559B2 (en) 2000-06-21 2017-07-18 Bioarray Solutions, Ltd. Multianalyte molecular analysis using application-specific random particle arrays
JP2003012548A (en) * 2001-06-27 2003-01-15 Japan Science & Technology Corp Pharmaceutical composition
CN100494395C (en) 2001-10-15 2009-06-03 生物芯片技术有限公司 polymorphic locus multiplexed analysis by concurrent probing and enzyme-mediated detection
WO2005042763A2 (en) 2003-10-28 2005-05-12 Bioarray Solutions Ltd. Optimization of gene expression analysis using immobilized capture probes

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0558944A2 (en) * 1992-02-06 1993-09-08 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA and DNA molecules for producing virus resistance
WO1995031541A2 (en) * 1994-05-18 1995-11-23 Ribozyme Pharmaceuticals, Inc. Methods and compositions for treatment of restenosis and cancer using ribozymes
WO1996010080A1 (en) * 1994-09-29 1996-04-04 Hybridon, Inc. Finderons and methods of their preparation and use
WO1996034099A2 (en) * 1995-04-28 1996-10-31 Hsc Research And Development Limited Partnership Genetic sequences and proteins related to alzheimer's disease, and uses therefor
WO1997043404A2 (en) * 1996-05-13 1997-11-20 Hybridon, Inc. Ribozyme variants with improved catalytic activity under low magnesium conditions
WO1997046678A1 (en) * 1996-06-06 1997-12-11 Bayer Corporation Nucleic acids and polypeptides related to presenilin
WO1998058057A1 (en) * 1997-06-19 1998-12-23 Innovir Laboratories, Inc. Compositions inducing cleavage of rna motifs

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0558944A2 (en) * 1992-02-06 1993-09-08 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA and DNA molecules for producing virus resistance
WO1995031541A2 (en) * 1994-05-18 1995-11-23 Ribozyme Pharmaceuticals, Inc. Methods and compositions for treatment of restenosis and cancer using ribozymes
WO1996010080A1 (en) * 1994-09-29 1996-04-04 Hybridon, Inc. Finderons and methods of their preparation and use
WO1996034099A2 (en) * 1995-04-28 1996-10-31 Hsc Research And Development Limited Partnership Genetic sequences and proteins related to alzheimer's disease, and uses therefor
WO1997043404A2 (en) * 1996-05-13 1997-11-20 Hybridon, Inc. Ribozyme variants with improved catalytic activity under low magnesium conditions
WO1997046678A1 (en) * 1996-06-06 1997-12-11 Bayer Corporation Nucleic acids and polypeptides related to presenilin
WO1998058057A1 (en) * 1997-06-19 1998-12-23 Innovir Laboratories, Inc. Compositions inducing cleavage of rna motifs

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
LEVY-LAHAD, E. ET AL.: "Candidate gene for the chromosome 1 familial Alzheimer's Disease locus", SCIENCE, vol. 269, 18 August 1995 (1995-08-18), pages 973 - 977, XP000616667 *
SCHMIDT, S. ET AL.: "Base and sugar requirements for RNA cleavage of essential nucleoside residues in internal loop B of the hairpin ribozyme: implications for secondary structure", NUCLEIC ACIDS RESEARCH, vol. 24, no. 4, 1996, pages 573 - 581, XP002129026 *
VITO, P. ET AL.: "Requirement of the familial Alzheimer's Disease gene PS2 for apoptosis", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 271, no. 49, 6 December 1996 (1996-12-06), pages 31025 - 31028, XP002097004 *

Cited By (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8691594B2 (en) 1996-04-25 2014-04-08 Bioarray Solutions, Ltd. Method of making a microbead array with attached biomolecules
US7056746B2 (en) 1996-04-25 2006-06-06 Bioarray Solutions Ltd. Array cytometry
US7144119B2 (en) 1996-04-25 2006-12-05 Bioarray Solutions Ltd. System and method for programmable illumination pattern generation
US7291504B2 (en) 1996-04-25 2007-11-06 Bioarray Solutions Ltd. Assay involving detection and identification with encoded particles
US7427512B2 (en) 1996-04-25 2008-09-23 Bioarray Solutions Ltd. Light-controlled electrokinetic assembly of particles near surfaces
US7041510B2 (en) 1996-04-25 2006-05-09 Bioarray Solutions Ltd. System and method for programmable illumination pattern generation
US9400259B2 (en) 1996-04-25 2016-07-26 Bioarray Solutions, Ltd. Method of making a microbead array with attached biomolecules
US9377388B2 (en) 1997-03-14 2016-06-28 Trustees Of Tufts College Methods for detecting target analytes and enzymatic reactions
US7057704B2 (en) 2000-09-17 2006-06-06 Bioarray Solutions Ltd. System and method for programmable illumination pattern generation
US9436088B2 (en) 2001-06-21 2016-09-06 Bioarray Solutions, Ltd. Un-supported polymeric film with embedded microbeads
US9251583B2 (en) 2002-11-15 2016-02-02 Bioarray Solutions, Ltd. Analysis, secure access to, and transmission of array images
US8691754B2 (en) 2003-09-22 2014-04-08 Bioarray Solutions, Ltd. Microparticles with enhanced covalent binding capacity and their uses
US9147037B2 (en) 2004-08-02 2015-09-29 Bioarray Solutions, Ltd. Automated analysis of multiplexed probe-target interaction patterns: pattern matching and allele identification

Also Published As

Publication number Publication date
EP1095138A2 (en) 2001-05-02
AR020329A1 (en) 2002-05-08
CA2332497A1 (en) 2000-01-20
AU5033999A (en) 2000-02-01
JP2002520016A (en) 2002-07-09
WO2000003004A2 (en) 2000-01-20

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