DE552178T1 - Abgeänderte ribozyme. - Google Patents

Abgeänderte ribozyme.

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Publication number
DE552178T1
DE552178T1 DE91916858T DE91916858T DE552178T1 DE 552178 T1 DE552178 T1 DE 552178T1 DE 91916858 T DE91916858 T DE 91916858T DE 91916858 T DE91916858 T DE 91916858T DE 552178 T1 DE552178 T1 DE 552178T1
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Prior art keywords
rna
group
modified
nucleotide
rna according
Prior art date
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Pending
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DE91916858T
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English (en)
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Fritz Benseler
Fritz Eckstein
David Olsen
Wolfgang Pieken
David Williams
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Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
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Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
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Publication of DE552178T1 publication Critical patent/DE552178T1/de
Pending legal-status Critical Current

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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • 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
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • 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
    • C12N2310/31—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
    • C12N2310/322—2'-R Modification

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
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  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
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  • Biochemistry (AREA)
  • AIDS & HIV (AREA)
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  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Communicable Diseases (AREA)
  • Veterinary Medicine (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Emulsifying, Dispersing, Foam-Producing Or Wetting Agents (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Transition And Organic Metals Composition Catalysts For Addition Polymerization (AREA)
  • Materials Applied To Surfaces To Minimize Adherence Of Mist Or Water (AREA)
  • Prostheses (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Claims (43)

Europäische Patentanmeldung Nr. 91 916 858. EU 2231 MAX-PLANCK-GESELLSCHAFT ZUR FÖRDERUNG DER WISSENSCHAFTEN E.V. Patentansprüche
1. RNA-Molekül mit katalytischer Aktivität, umfassend mindestens ein modifiziertes Nukleosid, worin die Hydroxylgruppe an der 2'-Position des Ribosezuckers durch eine Modifizierungsgruppe ersetzt ist, ausgewählt aus Halogen-, Sulfhydryl-, Azido-, Amino-, monosubstituierten Amino- und disubstituierten Aminogruppen.
2. RNA nach Anspruch 1, worin die Modifzierungsgruppe eine Halogen- oder eine Aminogruppe ist.
3. RNA nach Anspruch 1 oder 2, worin die Halogengruppe eine Fluorgruppe ist.
4. RNA nach Anspruch 1, worin die katalytische Aktivität mindestens eine aus der Gruppe umfaßt, bestehend aus Nukleotidyltransferase-, Dephosphorylase-, Deoxyribonuklease- und sequenzspezifischen Endoribonuklease-Aktivitäten.
5. RNA nach Anspruch 4, worin die katalytische Aktivität eine sequenzspezifische Endoribonuklease-Aktivität umfaßt.
6. RNA nach Anspruch 5, worin sie ein Hammerkopf-Ribozym oder eine Haarnadel-RNA ist.
7. RNA nach einem der vorhergehenden Ansprüche, worin die an den modifzierten Ribosezucker gebundene Nukleotidbase ausgewählt ist aus der Gruppe, bestehend aus natürlich in RNA vorkommenden Basen und substituierten Basen.
8. RNA nach Anspruch 7, worin die substituierte Nukleotidbase ausgewählt ist aus der Gruppe, bestehend aus Xanthin, Hypoxanthin, 2,6-Diaminopurin, 2-Hydroxy-6-mercaptopurin und Purinbasen, die an der Position 6 mit Schwefel substituiert sind, oder Pyrimidinbasen, die an der Position 5 mit Halogen- oder C1 -C5-Alkylgruppen substituiert sind.
9. RNA nach Anspruch 7, worin die an den modifizierten Ribosezucker gebundene Nukleotidbase eine natürlich in RNA vorkommende Base ist.
10. RNA nach Anspruch 9, worin die an den modifizierten Ribosezucker gebundene Nukleotidbase eine Pyrimidinbase ist.
11. RNA nach einem der vorhergehenden Ansprüche, worin alle Nukleotidbasen einer spezifischen Art an einen modifizierten Ribosezucker gebunden sind.
12. RNA nach Anspruch 11, worin alle Uracil-Nukleotidbasen an einen modifizierten Ribosezucker gebunden sind.
13. RNA nach Anspruch 11, worin alle Cytosin-Nukleotidbasen an einen modifizierten Ribosezucker gebunden sind.
14. RNA nach einem der Ansprüche 1 bis 10, worin alle Nukleotidbasen von zwei spezifischen Arten an einen modifizierten Ribosezucker gebunden sind.
15. RNA nach Anspruch 14, worin alle Cytosin- und Uracil-Nukleotidbasen an einen modifizierten Zucker gebunden sind.
16. RNA nach einem der Ansprüche 11 bis 15, worin der modifizierte Ribosezucker eine 2'-Fluor- oder 2'-Aminogruppe umfaßt.
17. RNA, umfassend die Nukleotidseguenz E2:
5'-GGG(2'-FU)CC(2'-FU)C(2'-FU)GA(2'-FU)GAGGCCG (2'-FU)(2'-FU)AGGCCGAAAC(2'-FU)CC-3', worin 2'-FU 2'-Deoxy-2'-fluoruridinmonophosphat bedeutet.
18. RNA, umfassend die Nukleotidseguenz E3:
5'-GGG^'-NH2 U)CC^-NH2 U)C (2'-NH2 U)GA(^-NH2U) GAGGCCG(2'-NH2 U)(2'-NH2 U)AGGCCGAAAC(2'-NH2 U)CC-3', worin 2'-NH2 U 2'-Deoxy-2'-aminouridinmonophosphat bedeutet.
19. RNA nach einem der Ansprüche 1 bis 10, umfassend ein selektives Modifikationsmuster auf Basis der strukturellen Charakteristiken des Moleküls.
20. RNA nach Anspruch 19, worin die Nukleotide an hypersensitiven Stellen für Ribonukleasen modifiziert sind.
21. RNA nach einem der vorhergehenden Ansprüche, weiterhin umfassend mindestens eine modifizierte internukleotidische Phosphodiesterbindung.
22. RNA nach Anspruch 21, worin die modifizierte Phosphodiesterbindung eine Phosphorthioatgruppe ist.
23. RNA nach Anspruch 21 oder 22, worin mindestens die 5'-terminale Phosphodiesterbindung modifiziert ist.
24. RNA nach einem der Ansprüche 21 bis 23, worin mindestens die 3'-terminale Phosphodiesterbindung modifiziert ist.
25. RNA nach einem der Ansprüche 21 bis 24, worin die 5'-terminale Phosphodiesterbindung und mindestens drei
3'-terminale Phosphodiesterbindungen modifiziert
sind.
26. Verfahren zur Synthese eines RNA-Moleküls mit katalytischer Aktivität, umfassend:
Einbauen von mindestens einem modifizierten Nukleotid in eine RNA-Kette, worin die Hydroxylgruppe an der 2'-Position des Ribosezuckers durch eine Modifizierungsgruppe ersetzt wird, ausgewählt aus Halogen-, SuIfhydryl-, Azido-, Amino-, monosubstituierten Amino- und
disubstituierten Aminogruppen.
27. Verfahren nach Anspruch 26, worin die Modifizierungsgruppe eine Halogen- oder eine Aminogruppe ist.
28. Verfahren nach Anspruch 26 oder 27, worin die Halogengruppe eine Fluorgruppe ist.
29. Verfahren nach Anspruch 27 oder 28, worin die Synthese der RNA-Kette durch chemische Synthese aus Nukleotidvorstufen auf einem festen Träger, Entfernen des RNA-Produkts vom festen Träger und Reinigen des entfernten RNA-Produkts durchgeführt wird.
30. Verfahren nach Anspruch 27 oder 28, worin die Synthese der RNA-Kette durch chemische Synthese aus Nukleotidvorstufen in Lösung und Reinigen des RNA-Produkts erfolgt.
31. Verfahren nach Anspruch 29 oder 30, worin die jeweiligen Phosphoramidite oder H-Phosphonate als Nukleotidvorstufen verwendet werden.
32. Verfahren nach einem der Ansprüche 29 bis 31, worin die Aminomodifizierungsgruppe in eine RNA-Kette in Form einer Trifluoracetylamidgruppe eingebaut wird und die Trifluoracetylschutzgruppe anschließend durch Behandlung mit Ammoniak entfernt wird.
33. Verfahren nach einem der Ansprüche 26 bis 32, weiterhin umfassend:
Einbauen von mindestens einer modifizierten internukleotidischen Phosphodiesterbindung in eine RNA-Kette.
34. Verfahren nach Anspruch 33, worin die modifizierte Phosphodiesterbindung eine Phosphorthioatgruppe ist.
35. Verfahren nach einem der Ansprüche 26 bis 28, worin die Synthese der RNA-Kette durch Transkription von einem Nukleinsäuretemplate durch eine Nukleinsäurepolymerase erfolgt.
36. Verfahren nach Anspruch 35, worin das Nukleinsäuretemplate ein DNA-Template ist und die Nukleinsäurepolymerase eine DNA-abhängige RNA-Polymerase ist.
37. Verfahren nach Anspruch 36, worin die DNA-abhängige RNA-Polymerase aus der Gruppe, bestehend aus T7-, T3- und SP6-Polymerase, ausgewählt wird.
38. Verfahren nach Anspruch 37, worin die DNA-abhängige RNA-Polymerase T7-Polymerase ist.
39. Verfahren nach einem der Ansprüche 35 bis 38, worin die Modifizierungsgruppe eine Halogengruppe ist und die Synthese der RNA-Kette in Gegenwart von Mn2+-Ionen durchgeführt wird.
40. Verfahren nach einem der Ansprüche 35 bis 38, worin die Modifizierungsgruppe eine Amino-, monosubstituierte Amino- oder disubstituierte Aminogruppe ist und die Synthese der RNA-Kette in Gegenwart von Mg2 + -Ionen durchgeführt wird.
41. Verwendung einer RNA nach einem der Ansprüche 1 bis als therapeutisches Mittel oder Biokatalysator.
42. Therapeutisches Mittel, umfassend als Wirkstoff eine RNA nach einem der Ansprüche 1 bis 25 gegebenenfalls zusammen mit gebräuchlichen Füllmitteln, Hilfsmitteln, Trägermitteln und Verdünnungsmitteln.
43. Verfahren zur Herstellung eines therapeutischen Mittels, worin das therapeutische Mittel als Wirkstoff eine RNA nach einem der Ansprüche 1 bis 25, gegebenenfalls mit üblichen Füllmitteln, Hilfsmitteln, Trägermitteln und Verdünnungsmitteln enthält.
DE91916858T 1990-10-12 1991-09-23 Abgeänderte ribozyme. Pending DE552178T1 (de)

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US (3) US5672695A (de)
EP (1) EP0552178B1 (de)
JP (2) JP3257675B2 (de)
AT (1) ATE147098T1 (de)
AU (1) AU649074B2 (de)
CA (1) CA2093664C (de)
DE (2) DE69123979T2 (de)
ES (1) ES2061416T3 (de)
GR (1) GR930300130T1 (de)
PL (1) PL169576B1 (de)
WO (1) WO1992007065A1 (de)

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