CA2207593A1 - Procede et reactif servant a traiter les etats arthritiques, a introduitre une tolerance aux greffes et a inverser les reactions immunes - Google Patents

Procede et reactif servant a traiter les etats arthritiques, a introduitre une tolerance aux greffes et a inverser les reactions immunes

Info

Publication number
CA2207593A1
CA2207593A1 CA002207593A CA2207593A CA2207593A1 CA 2207593 A1 CA2207593 A1 CA 2207593A1 CA 002207593 A CA002207593 A CA 002207593A CA 2207593 A CA2207593 A CA 2207593A CA 2207593 A1 CA2207593 A1 CA 2207593A1
Authority
CA
Canada
Prior art keywords
nucleic acid
rna
ribozyme
chemically reactive
group
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.)
Abandoned
Application number
CA002207593A
Other languages
English (en)
Inventor
John Gustofson
Nassim Usman
Alex Burgin
Leonid Beigelman
Anil Modak
Francine Wincott
Kenneth Draper
Alexander Karpeisky
James D. Thompson
James Mcswiggen
Pamela Pavco
Daniel T. Stinchcomb
Thale Jarvis
Jasenka Matulic-Adamic
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.)
Sirna Therapeutics Inc
Original Assignee
Individual
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
Priority claimed from US08/363,253 external-priority patent/US5891683A/en
Priority claimed from US08/363,254 external-priority patent/US5891684A/en
Priority claimed from US08/390,850 external-priority patent/US5612215A/en
Priority claimed from US08/426,124 external-priority patent/US5716824A/en
Priority claimed from US08/432,874 external-priority patent/US5627053A/en
Priority claimed from US08/512,861 external-priority patent/US6146886A/en
Priority claimed from US08/541,365 external-priority patent/US5672501A/en
Application filed by Individual filed Critical Individual
Publication of CA2207593A1 publication Critical patent/CA2207593A1/fr
Abandoned legal-status Critical Current

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    • 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
    • C12N15/1137—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 enzymes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
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    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
    • C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/24—Metalloendopeptidases (3.4.24)
    • C12Y304/24017—Stromelysin 1 (3.4.24.17)
    • 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
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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
    • C12N2310/121—Hammerhead
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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

Abstract

Molécule d'acide nucléique enzymatique effectuant le clivage de l'ARN associé à l'évolution ou à l'existence d'un état arthritique, à l'introduction d'une tolérance aux greffes ou à l'inversion d'une réaction immune. En particulier, les séquences de ribozymes sont dirigées vers un ARNm codant B7-1, B7-3, cd40 et/ou stromelysine. L'invention concerne également des ribozymes où uracil des positions 4 et/ou 7 est substitué, ainsi que des procédés de synthèse de 2'-alkylnucléotides, 2'-O-alkylthioalkyle ou 2'-alkylnucléotides, 2'-O-alkylthioalkyle ou 2'-alkylthioalkylnucléotides. Elle concerne, de plus, un procédé de déprotection de l'ARN par éthylamine aqueux, un procédé de synthèse de produits mimétiques de ribonucléosides de base, ainsi que des unités de transcription composés d'un promoteur de polymérase II d'ARN, d'un promoteur nucléaire réduit de U6 ou d'un système promoteur de VA1 d'adénovirus.
CA002207593A 1994-12-13 1995-11-22 Procede et reactif servant a traiter les etats arthritiques, a introduitre une tolerance aux greffes et a inverser les reactions immunes Abandoned CA2207593A1 (fr)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US35492094A 1994-12-13 1994-12-13
US08/354,920 1994-12-13
US08/363,253 1994-12-23
US08/363,254 1994-12-23
US08/363,253 US5891683A (en) 1993-09-02 1994-12-23 Non-nucleotide containing enzymatic nucleic acid
US08/363,254 US5891684A (en) 1992-10-15 1994-12-23 Base-modified enzymatic nucleic acid
US08/390,850 US5612215A (en) 1992-12-07 1995-02-17 Stromelysin targeted ribozymes
US08/390,850 1995-02-17
US08/426,124 1995-04-20
US08/426,124 US5716824A (en) 1995-04-20 1995-04-20 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US08/432,874 US5627053A (en) 1994-03-29 1995-05-02 2'deoxy-2'-alkylnucleotide containing nucleic acid
US08/432,874 1995-05-02
US43450995A 1995-05-04 1995-05-04
US08/434,509 1995-05-04
US60/000,974 1995-07-07
US60/000,951 1995-07-07
US08/512,861 US6146886A (en) 1994-08-19 1995-08-07 RNA polymerase III-based expression of therapeutic RNAs
US08/512,861 1995-08-07
US08/541,365 US5672501A (en) 1994-12-23 1995-10-05 Base-modified enzymatic nucleic acid
US08/541,365 1995-10-05

Publications (1)

Publication Number Publication Date
CA2207593A1 true CA2207593A1 (fr) 1996-06-20

Family

ID=56289667

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002207593A Abandoned CA2207593A1 (fr) 1994-12-13 1995-11-22 Procede et reactif servant a traiter les etats arthritiques, a introduitre une tolerance aux greffes et a inverser les reactions immunes

Country Status (3)

Country Link
AU (1) AU4412096A (fr)
CA (1) CA2207593A1 (fr)
WO (1) WO1996018736A2 (fr)

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