DK2363479T3 - Oligoribonukleotid til inhibering af ekspressionen af et defineret gen - Google Patents
Oligoribonukleotid til inhibering af ekspressionen af et defineret gen Download PDFInfo
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- DK2363479T3 DK2363479T3 DK10011217.6T DK10011217T DK2363479T3 DK 2363479 T3 DK2363479 T3 DK 2363479T3 DK 10011217 T DK10011217 T DK 10011217T DK 2363479 T3 DK2363479 T3 DK 2363479T3
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Claims (14)
1. Oligoribonukleotid med dobbeltstrenget struktur (dsRNA) til inhibering af ekspressionen af et defineret målgen i pattedyrceller, hvordsRNA’et udviser 15 til 49 basepar, og en streng af dsRNA’et udviser et område I, der i det mindste afsnitsvis er komplementært til målgenet og udviser højst 49 efter hinanden følgende nukleotidpar, og et inden for den dobbeltstrengede struktur komplementært område II er dannet af to separate RNA-enkeltstrenge, hvor nukleotiderne af dsRNA’et er modficerede, hvor mindst en 2’-hydroxylgruppe af nukleotiderne af dsRNA’et i det komplementære område II er erstattet af en kemisk gruppe.
2. Medikament med mindst et oligoribonukleotid med 15 til 49 basepar og med dobbeltstrenget struktur (dsRNA) til inhibering af ekspressionen af et defineret målgen i pattedyrceller, hvor en streng af dsRNA’et udviser et område I, der i det mindste afsnitsvis er komplementært til målgenet og udviser højst 49 efter hinanden følgende nukleotidpar, og et inden for den dobbeltstrengede struktur komplementært område II er dannet af to separate RNA-enkeltstrenge, hvor nukleotiderne af dsRNA’et er modficerede, hvor mindst en 2’-hydroxylgruppe af nukleotiderne af dsRNA’et i det komplementære område II er erstattet afen kemisk gruppe.
3. Anvendelse af et oligonukleotid med 15 til 49 basepar og med dobbeltstrenget struktur (dsRNA) til fremstilling af et medikament til inhibering af ekspressionen af et defineret målgen i pattedyrceller, hvor en streng af dsRNA’et udviser et område I, der i det mindste afsnitsvis er komplementært til målgenet og udviser højst 49 efter hinanden følgende nukleotidpar, og et inden for den dobbeltstrengede struktur komplementært område II er dannet af to separate RNA-enkeltstrenge, hvor nukleotiderne af dsRNA’et er modficerede, hvor mindst en 2’-hydroxylgruppe af nukleotiderne af dsRNA’et i det komplementære område II er erstattet af en kemisk gruppe.
4. DsRNA ifølge krav 1, medikament ifølge krav 2 eller anvendelse ifølge krav 3, hvor dsRNA’et foreligger pakket i micellare strukturer, fortrinsvis i liposomer.
5. DsRNA, medikament eller anvendelse ifølge et af de foregående krav, hvor dsRNA’et er indesluttet i virale naturlige kapsider eller er indesluttede i på kemisk eller enzymatisk måde fremstillede kunstige kapsider eller deraf afledte strukturer.
6. DsRNA, medikament eller anvendelse ifølge et af de foregående krav, hvor målgenet er en bestanddel af et virus.
7. DsRNA, medikament eller anvendelse ifølge krav 6, hvor viruset er et humanpatogent eller dyrepatogent virus.
8. DsRNA, medikament eller anvendelse ifølge et af de foregående krav, hvor enderne af dsRNA’et er modificerede for at modvirke en nedbrydelse i pattedyrcellerne eller en dissociation i enkeltstrengene.
9. DsRNA, medikament eller anvendelse ifølge et af de foregående krav, hvor den af nukleotidparrene fremkaldte sammenhæng af det komplementære område II er forøget af mindst en, fortrinsvis to, yderligere kemiske koblinger.
10. DsRNA, medikament eller anvendelse ifølge krav 9, hvor den kemiske kobling er dannet af en kovalent eller ionisk binding, en hydrogenbrobinding, hydrofobe vekselvirkninger, fortrinsvis van der Waals- eller stabelvekselvirkninger eller ved metal-ionkoordination.
11. DsRNA, medikament eller anvendelse ifølge krav 9 eller 10, hvor den kemiske kobling er frembragt ved mindst en ende, fortrinsvis ved begge ender af det komplementære område II.
12. DsRNA, medikament eller anvendelse ifølge et af de foregående krav, hvor dsRNA’et er bundet til, associeret med eller omgivet af mindst et fra et virus stammende, deraf afledt eller et syntetisk fremstillet viralt kappeprotein.
13. DsRNA, medikament eller anvendelse ifølge krav 12, hvor den ene side ved dannelsen af et kapsid eller en kapsidlignende struktur fra kappeproteinet vender mod det indre af kapsidet eller den kapsidlignende struktur.
14. DsRNA, medikament eller anvendelse ifølge et af de foregående krav, hvor pattedyrcellerne er menneskeceller.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DE19903713 | 1999-01-30 | ||
| DE19956568A DE19956568A1 (de) | 1999-01-30 | 1999-11-24 | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
| EP06025389.5A EP1798285B1 (de) | 1999-01-30 | 2000-01-29 | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
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| DK2363479T3 true DK2363479T3 (da) | 2016-10-31 |
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| DK02003683.6T DK1214945T4 (da) | 1999-01-30 | 2000-01-29 | Fremgangsmåde og medikament til at hæmme ekspressionen af et defineret målgen |
| DK10011217.6T DK2363479T3 (da) | 1999-01-30 | 2000-01-29 | Oligoribonukleotid til inhibering af ekspressionen af et defineret gen |
| DK05002454T DK1550719T3 (da) | 1999-01-30 | 2000-01-29 | Dobbeltstrenget RNA (dsRNA) til inhibering af ekspressionen af et defineret gen |
| DK06025389.5T DK1798285T3 (da) | 1999-01-30 | 2000-01-29 | Fremgangsmåde og medikament til hæmning af ekspressionen af et givent gen |
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| DK02003683.6T DK1214945T4 (da) | 1999-01-30 | 2000-01-29 | Fremgangsmåde og medikament til at hæmme ekspressionen af et defineret målgen |
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| Application Number | Title | Priority Date | Filing Date |
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| DK05002454T DK1550719T3 (da) | 1999-01-30 | 2000-01-29 | Dobbeltstrenget RNA (dsRNA) til inhibering af ekspressionen af et defineret gen |
| DK06025389.5T DK1798285T3 (da) | 1999-01-30 | 2000-01-29 | Fremgangsmåde og medikament til hæmning af ekspressionen af et givent gen |
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2000
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