DK1798285T3 - Fremgangsmåde og medikament til hæmning af ekspressionen af et givent gen - Google Patents
Fremgangsmåde og medikament til hæmning af ekspressionen af et givent gen Download PDFInfo
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- DK1798285T3 DK1798285T3 DK06025389.5T DK06025389T DK1798285T3 DK 1798285 T3 DK1798285 T3 DK 1798285T3 DK 06025389 T DK06025389 T DK 06025389T DK 1798285 T3 DK1798285 T3 DK 1798285T3
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Claims (26)
1. Oligoribonukleotid med dobbeltstrenget struktur (dsRNA) til hæmning af ekspressionen af et givet målgen i pattedyrsceller, idet dsRNA'et består af 15 til 49 basepar, og en streng i dsRNA'et har et område I, som er komplementært til målgenet og består af højst 49 på hinanden følgende nukleotidpar, og et inden for den dobbeltstrengede struktur komplementært område II, der er dannet af to separate RNA-enkeltstrenge, idet oligoribonukleotidet i alt har en ved hjælp af område I defineret nukleotidsekvens, idet område I har den samme længde som dsRNA'et, og idet dsRNA'et foreligger pakket i micellære strukturer, fortrinsvis i liposomer.
2. DsRNA ifølge krav 1, idet målgenet er udvalgt af den følgende gruppe: onkogen, cytokin-gen, Id-protein-gen, udviklingsgen, PKR-gen, priongen.
3. DsRNA ifølge et af de foregående krav, idet dsRNA'et er indesluttet i virale naturlige capsider eller i kunstige capsider fremstillet på kemisk eller enzymatisk måde eller heraf afledte strukturer.
4. DsRNA ifølge et af de foregående krav, idet målgenet er bestanddel af et virus.
5. DsRNA ifølge krav 4, idet virusset er et humanpatogent virus.
6. DsRNA ifølge krav 4, idet virusset er et animalsk patogent virus.
7. DsRNA ifølge et af de foregående krav, idet enderne på dsRNA'et er modificeret for at modvirke en nedbrydning i pattedyrscellerne eller en dissociation i enkeltstrengene.
8. DsRNA ifølge et af de foregående krav, idet den ved hjælp af nukleotidparrene bevirkede sammenholdning af det komplementære område II er forøget ved hjælp af i det mindste en, fortrinsvis to, yderligere kemiske bindinger.
9. DsRNA ifølge krav 8, idet den kemiske binding er dannet ved hjælp af en kovalent eller ionisk binding, en hydrogenbrobinding, hydrofobe vekselvirkninger, fortrinsvis Van-der-Waals- eller staplingsvekselvirkninger, eller ved hjælp af metal-ionkoordination.
10. DsRNA ifølge krav 8 eller 9, idet den kemiske binding er fremstillet på den i det mindste ene, fortrinsvis på begge ender, af det komplementære område II.
11. DsRNA ifølge et af kravene 8 til 10, idet den kemiske binding er dannet ved hjælp af en eller flere forbindelsesgrupper, idet forbindelsesgrupperne fortrinsvis er poly-(oxyphosphinicooxy-1,3-propandiol)- og/eller polyethylenglycol-kæder.
12. DsRNA ifølge et af kravene 8 til 10, idet den kemiske binding er dannet ved hjælp af i de komplementære områder II i stedet for puriner anvendte purinanaloga.
13. DsRNA ifølge et af kravene 8 til 10, idet den kemiske binding er dannet ved hjælp af azabenzenenheder indkoblet i de komplementære områder II.
14. DsRNA ifølge et af kravene 8 til 10, idet den kemiske binding er dannet ved hjælp af i de komplementære områder II i stedet for nukleotider anvendte forgrenede nukleotidanaloga.
15. DsRNA ifølge et af kravene 8 til 10, idet der til fremstilling af den kemiske binding benyttes i det mindste en af de følgende grupper: methylenblåt; bifunktionelle grupper, fortrinsvis bis-(2-chlorethyl)-amin; N-acetyl-N'-(p-glyoxyl-benzoyl)-cystamin; 4-thiouracil; psoralen.
16. DsRNA ifølge et af kravene 8 til 10, idet den kemiske binding er dannet ved hjælp af thiophosphoryl-grupper tilvejebragt på enderne af det dobbeltstrengede område.
17. DsRNA ifølge et af kravene 8 til 10, idet den kemiske binding på enderne af det dobbeltstrengede område er tilvejebragte tripelhelix-bindinger.
18. DsRNA ifølge et af de foregående krav, idet nukleotiderne i dsRNA'et er modificeret.
19. DsRNA ifølge et af de foregående krav, idet i det mindste en 2'-hydroxylgruppe i nukleotiderne i dsRNA'et i det komplementære område II er erstattet af en kemisk gruppe, fortrinsvis en 2'-amino- eller en 2'-methylgruppe.
20. DsRNA ifølge et af de foregående krav, idet i det mindste et nukleotid i i det mindste en streng i det komplementære område II er et "locked nucleotide" med en fortrinsvis ved hjælp af en 2'-O, 4'-C-methylenbo kemisk modificeret sukkerring.
21. DsRNA ifølge et af de foregående krav, idet dsRNA'et er bundet til i det mindste fra et virus stammende, herfra afledt eller et syntetisk fremstillet viralt kapselprotein, er associeret hermed eller er omgivet heraf.
22. DsRNA ifølge krav 21, idet kapselproteinet af afledt af polyomavirusset.
23. DsRNA ifølge krav 21 eller 22, idet kapselproteinet indeholder virus-proteinet 1 (VP1) og/eller virus-proteinet 2 (VP2) i polyomavirusset.
24. DsRNA ifølge et af kravene 21 til 23, idet den ene side ved dannelsen af et capsid eller et capsidagtigt produkt ud fra kapselproteinet er vendt mod det indre af capsidet eller den capsidagtige dannelse.
25. DsRNA ifølge et af de foregående krav, idet dsRNA'et er komplementært med det primære eller processerede RNA- transkript af målgenet.
26. DsRNA ifølge et af de foregående krav, idet pattedyrscellerne er menneskelige celler.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
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DE19903713 | 1999-01-30 | ||
DE19956568A DE19956568A1 (de) | 1999-01-30 | 1999-11-24 | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
EP05002454A EP1550719B1 (de) | 1999-01-30 | 2000-01-29 | Doppelsträngige RNA (dsRNA) zur Hemmung der Expression eines vorgegebenen Gens |
Publications (1)
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DK1798285T3 true DK1798285T3 (da) | 2017-07-03 |
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Family Applications (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK05002454T DK1550719T3 (da) | 1999-01-30 | 2000-01-29 | Dobbeltstrenget RNA (dsRNA) til inhibering af ekspressionen af et defineret gen |
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 |
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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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 |
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 |
Country Status (14)
Country | Link |
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US (17) | US8101584B2 (da) |
EP (6) | EP1144623B9 (da) |
JP (14) | JP2003502012A (da) |
AT (3) | ATE418610T1 (da) |
AU (2) | AU778474B2 (da) |
CA (1) | CA2359180C (da) |
CY (3) | CY1108896T1 (da) |
DE (7) | DE19956568A1 (da) |
DK (4) | DK1550719T3 (da) |
ES (5) | ES2321409T3 (da) |
HK (1) | HK1160487A1 (da) |
PT (4) | PT1214945E (da) |
WO (1) | WO2000044895A1 (da) |
ZA (1) | ZA200105909B (da) |
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DE19956568A1 (de) | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
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1999
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2000
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- 2000-01-29 DK DK05002454T patent/DK1550719T3/da active
- 2000-01-29 ES ES06025389.5T patent/ES2628535T3/es not_active Expired - Lifetime
- 2000-01-29 DK DK02003683.6T patent/DK1214945T4/da active
- 2000-01-29 ES ES00910510T patent/ES2182791T3/es not_active Expired - Lifetime
- 2000-01-29 EP EP00910510A patent/EP1144623B9/de not_active Revoked
- 2000-01-29 AT AT05002454T patent/ATE418610T1/de active
- 2000-01-29 DE DE20023125U patent/DE20023125U1/de not_active Expired - Lifetime
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- 2000-01-29 DK DK10011217.6T patent/DK2363479T3/da active
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