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DK2258847T3 - Yderligere nye former af interferende RNA-molekyler - Google Patents

Yderligere nye former af interferende RNA-molekyler Download PDF

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Publication number
DK2258847T3
DK2258847T3 DK10012263.9T DK10012263T DK2258847T3 DK 2258847 T3 DK2258847 T3 DK 2258847T3 DK 10012263 T DK10012263 T DK 10012263T DK 2258847 T3 DK2258847 T3 DK 2258847T3
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Prior art keywords
misc
feature
nucleotide
strand
rnai
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DK10012263.9T
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English (en)
Inventor
Klaus Giese
Jörg Kaufmann
Anke Klippel-Giese
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Silence Therapeutics Gmbh
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=31720998&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK2258847(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from EP02017601A external-priority patent/EP1389637B1/en
Application filed by Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
Application granted granted Critical
Publication of DK2258847T3 publication Critical patent/DK2258847T3/da

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Claims (14)

1. Ribonukleinsyre egnet til at mediere RNA-interferens omfattende en dobbeltstrenget struktur, hvor den dobbeltstrengede struktur omfatter en første streng og en anden streng, hvor den første streng omfatter et første stykke af sammenhængende nukleotider og hvor det første stykke er mindst delvist komplementært til en målnukleinsyre, og den anden streng omfatter et andet stykke af sammenhængende nukleotider og hvor det andet stykke er mindst delvist identisk med målnukleinsyren, kendetegnet ved at den første streng og den anden streng omfatter en flerhed af grupper af modificerede nukleotider, der haren modifikation ved 2'-positionen, hvor hver gruppe af modificerede nukleotider i strengen er flankeret på en eller begge sider afen flankeringsgruppe af nukleotider, hvor flankeringsnukleotidet/-nukleotiderne, der danner flankeringsgruppen af nukleotider enten er et umodificeret nukleotid(er) elleret nukleotid(er), der haren modifikation forskellig fra modifikationen af de modificerede nukleotider, den første streng eller første stykke og den anden streng eller andet stykke danner et mønster af modificerede nukleotider, hvor grupper af modificerede nukleotider er skiftevis med flankeringsgrupper af nukleotider og mønsteret af modificerede nukleotider af den første streng er det samme som mønsteret af modificerede nukleotider af den anden streng.
2. Ribonukleinsyren ifølge krav 1, hvor modifikationen er valgt fra gruppen omfattende amino, fluor, methoxy, alkoxy og alkyl.
3. Ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 2, hvor den dobbeltstrengede struktur er stump-endet.
4. Ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 3, hvor længden af den dobbeltstrengede struktur har en længde fra omkring 17 til 21 og mere fortrinsvis 18 eller 19 baser.
5. Ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 4, hvor længden af den første streng og/eller længden af den anden streng er uafhængigt af hinanden valgt fra gruppen omfattende områderne fra omkring 15 til omkring 23 baser, 17 til 21 baser og 18 eller 19 baser.
6. Ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 5, hvor både den første streng og den anden streng hver omfatter mindst en gruppe af modificerede nukleotider og mindst en flankeringsgruppe af nukleotider, hvor hver gruppe af modificerede nukleotider omfatter mindst et nukleotid og hvor hver flankeringsgruppe af nukleotider omfatter mindst et nukleotid; hvor hver gruppe af modificerede nukleotider af den første streng er linjestillet (aligned) med en flankeringsgruppe af nukleotider på den anden streng, hvor den mest terminale 5' nukleotid af den første streng er et nukleotid af gruppen af modificerede nukleotider, og den mest terminale 3' nukleotid af den anden streng er et nukleotid af flankeringsgruppen af nukleotider.
7. Ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 2 og 4 til 6, hvor den første streng og den anden streng er forbundet af en sløjfestruktur.
8. Ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 7 til anvendelse i en fremgangsmåde til behandlingen afen sygdom.
9. Anvendelse afen ribonukleinsyre ifølge et hvilket som helst af kravene 1 til 7, til målvalidering.
10. Anvendelse af en ribonukleinsyre ifølge et hvilket som helst af kravene 1 til 7 til fremstillingen af et medikament, hvor fortrinsvis medikamentet er til behandlingen af en sygdom eller af en tilstand, der er valgt fra gruppen omfattende glioblastom, prostatacancer, brystcancer, lungecancer, levercancer, koloncancer, pankreascancer og leukæmi, diabetes, fedme, kardiovaskulære sygdomme, og metaboliske sygdomme.
11. Celle, fortrinsvis en knockdown-celle, der indeholder en ribonukleinsyre ifølge et hvilket som helst af kravene 1 til 7, hvor, hvis cellen er en human celle, den humane celle er en isoleret human celle.
12. Organisme, fortrinsvis en knockdown-organisme, der indeholder en ribonukleinsyre ifølge et hvilket som helst af kravene 1 til 7, hvor organismen er forskellig fra et menneske.
13. Farmaceutisk sammensætning, der indeholder en ribonukleinsyre ifølge et hvilket som helst af kravene 1 til 7 og en farmaceutisk acceptabel bærer.
14. In w'tro-fremgangsmåde til inhibering af ekspressionen af et mål-gen i en celle eller derivat deraf omfattende at indføre en ribonukleinsyre ifølge et hvilket som helst af kravene 1 til 7 i cellen i en mængde tilstrækkelig til at inhibere ekspression af mål-genet, hvor mål-genet er mål-genet for ribonukleinsyren ifølge et hvilket som helst af kravene 1 til 7.
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