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DK1633767T3 - Fremgangsmåder og sammensætninger til styring af virkning af rna-silencing - Google Patents

Fremgangsmåder og sammensætninger til styring af virkning af rna-silencing Download PDF

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DK1633767T3
DK1633767T3 DK04753864.0T DK04753864T DK1633767T3 DK 1633767 T3 DK1633767 T3 DK 1633767T3 DK 04753864 T DK04753864 T DK 04753864T DK 1633767 T3 DK1633767 T3 DK 1633767T3
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rna
rnai agent
nucleotide
cell
rnai
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Phillip D Zamore
Guiliang Tang
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Univ Massachusetts
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Claims (27)

1. Anvendelsen af et RNAi-middel til at forstærke RNA-silencing af et mål-mRNA i en ikke-terapeutisk fremgangsmåde af RNA-silencing der er udført på en celle, der har en RNAi-signalvej; hvilket RNAi-middel har mindst et terminalt nucleotid, der er substitueret med et nucleotid, som danner et G:U-wobble-basepar med det tilsvarende nucleotid i mål-mRNA'et, hvor det terminale nucleotid er inden for 5 eller færre nucleotider fra 3'-enden af RNAi-midlet; hvilket RNAi-middel har mindst et terminalt nucleotid, der er substitueret med et nucleotid, som danner et G:U-wobble-basepar med det tilsvarende nucleotid i mål-mRNA'et, hvor det terminale nucleotid er inden for 5 eller færre nucleotider fra 5'-enden af RNAi-midlet; og hvor mindst to terminale nucleotider er substituerede, hvilke to terminale nucleotider er substitueret ved 3'-enden af RNAi-midlet, hvilken fremgangsmåde omfatter at bringe cellen i kontakt med RNAi-midlet under betingelser, således at silencing forstærkes.
2. Anvendelsen ifølge krav 1, hvor substitutionen er en A —> G-substitution, hvilket G danner et G:U-wobble-basepar med en U i det tilsvarende mål-mRNA.
3. Anvendelsen ifølge krav 1, hvor substitutionen er en C —> U-substitution, hvilket U danner et G:U-wobble-basepar med en G i det tilsvarende mål-mRNA.
4. Anvendelsen ifølge krav 1, hvor mindst tre, fire eller fem terminale nucleotider er substitueret.
5. Anvendelse ifølge et hvilket som helst af de foregående krav, hvor midlet syntetiseres kemisk.
6. Anvendelse ifølge et hvilket som helst af kravene 1-4, hvor midlet syntetiseres enzymatisk.
7. Anvendelse ifølge et hvilket som helst af de foregående krav, hvor midlet er afledt af et genteknisk fremstillet forstadium.
8. Anvendelse ifølge et hvilket som helst af de foregående krav, hvor cellen er en plantecelle.
9. Anvendelse ifølge et hvilket som helst af kravene 1-7, hvor cellen er en pattedyrcelle.
10. Anvendelse ifølge et hvilket som helst af de foregående krav, hvor RNAi-midlet er et Iille-hårnål -RNA (shRNA).
11. RNAi-middel til anvendelse til forstærkning af RNA-silencing af et mål-mRNA i en terapeutisk fremgangsmåde til RNA-silencing af et mål-RNA i et individ; hvor RNAi-midlet har mindst et terminalt nucleotid, der er substitueret med et nucleotid, som danner et G:U-wobble-basepar med det tilsvarende nucleotid i mål-mRNA'et, hvor det terminale nucleotid er inden for 5 eller færre nucleotider fra 3'-enden af RNAi-midlet; hvor RNAi-midlet har mindst et terminal nucleotid, der er substitueret med et nucleotid, som danner et G:U-wobble-basepar med det tilsvarende nucleotid i mål-mRNA'et, hvor det terminale nucleotid er inden for 5 eller færre nucleotider fra 5'-enden af RNAi-midlet; og hvor mindst to terminale nucleotider er substituerede, hvilke to terminale nucleotider er substitueret ved 3'-enden af RNAi-midlet, hvor fremgangsmåden omfatter administration til individet af en farmaceutisk sammensætning, der omfatter RNAi-midlet, således at silencing forstærkes.
12. RNAi-middel til anvendelsen ifølge krav 11, hvor substitutionen er en A —> G-substitution, hvilket G danner et G:U-wobble-basepar med en U i det tilsvarende mål-mRNA.
13. RNAi-middel til anvendelsen ifølge krav 11, hvor substitutionen er en C —> U-substitution, hvilket U danner et G:U-wobble-basepar med en G i det tilsva- rende mål-mRNA.
14. RNAi-middel til anvendelsen ifølge krav 11, hvor mindst tre, fire eller fem terminale nucleotider er substitueret.
15. RNAi-middel til anvendelse ifølge et hvilket som helst af kravene 11-14, hvor midlet syntetiseres kemisk.
16. RNAi-middel til anvendelse ifølge et hvilket som helst af kravene 11-14, hvor midlet syntetiseres enzymatisk.
17. RNAi-middel til anvendelse ifølge et hvilket som helst af kravene 11-14, hvor midlet er afledt af et genteknisk fremstillet forstadium.
18. RNAi-middel til anvendelse ifølge et hvilket som helst af kravene 11-17, hvor RNAi-midlet er et lille-hårnål-RNA (shRNA).
19. Ikke-terapeutisk fremgangsmåde til forstærkning af RNA-silingsaktivitet af et RNAi-middel, der omfatter: (i) at udvælge en mål-mRNA-sekvens; og (ii) at syntetisere et RNAi-middel, i hvilket mindst et terminalt nucleotid er substitueret med et nucleotid, som danner et G:U-wobble-basepar med det tilsvarende nucleotid i mål-mRNA'et, hvor det terminale nucleotid er inden for 5 eller færre nucleotider fra 3'-enden af RNAi-midlet; hvor RNAi-midlet har mindst et terminal nucleotid, der er substitueret med et nucleotid, der danner et G:U-wobble-basepar med det tilsvarende nucleotid i mål-mRNA'et, hvor det terminale nucleotid er inden for 5 eller færre nucleotider fra 5'-enden af RNAi-midlet; og hvor mindst to terminale nucleotider er substitueret, hvilke to terminale nucleotider er substitueret ved 3'-enden af RNAi-midlet.
20. Fremgangsmåden ifølge krav 19, hvor substitutionen er en A —> G-substitution, hvilket G danner et G:U-wobble-basepar med en U i det tilsvarende mål-mRNA.
21. Fremgangsmåden ifølge krav 19, hvor substitutionen er en C —> ll-substitution, hvilket U danner et G:U-wobble-basepar med en G i det tilsvarende mål-mRNA.
22. Fremgangsmåden ifølge krav 19, hvor mindst tre, fire eller fem terminale nucleotider er substitueret.
23. Fremgangsmåde ifølge et hvilket som helst af kravene 19-22, hvor midlet syntetiseres kemisk.
24. Fremgangsmåde ifølge et hvilket som helst af kravene 19-22, hvor midlet syntetiseres enzymatisk.
25. Fremgangsmåde ifølge et hvilket som helst af kravene 19-24, hvor midlet er afledt af et genteknisk fremstillet forstadium.
26. Fremgangsmåde ifølge et hvilket som helst af kravene 19-24, hvor RNAi-midlet er et Iille-hårnål-RNA (shRNA).
27. Anvendelse ifølge krav 1, hvor RNAi-midlet er til anvendelse ifølge krav 11 eller fremgangsmåden ifølge krav 19, hvor G:U-wobble-baseparet katalyserer selektiv spaltning af vildtype-mål-mRNA'et i forhold til spaltning af en mutant af vildtype-mål-mRNA’et.
DK04753864.0T 2003-06-02 2004-06-02 Fremgangsmåder og sammensætninger til styring af virkning af rna-silencing DK1633767T3 (da)

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