DK1888749T3 - Oligonukleotider til RNA-interferens samt biologiske anvendelser deraf - Google Patents

Oligonukleotider til RNA-interferens samt biologiske anvendelser deraf Download PDF

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DK1888749T3
DK1888749T3 DK06754634.1T DK06754634T DK1888749T3 DK 1888749 T3 DK1888749 T3 DK 1888749T3 DK 06754634 T DK06754634 T DK 06754634T DK 1888749 T3 DK1888749 T3 DK 1888749T3
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sequence
transfection
composition according
oligonucleotide
oligonucleotides
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DK06754634.1T
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Jean-Paul Behr
Bellemin Anne-Laure Bolcato
Patrick Erbacher
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Polyplus Transfection
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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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Claims (17)

1. Sammensætning til RNA-interferens omfattende dobbeltstrengede oligonukleotider med identiske eller forskellige sekvenser og/eller længder, idet oligonukleotiderne har sekvenserne 3N1N2...Ni_1Ni...Nj5', hvori - 3Ni...Nj5' er en 19-28mer antisense-sekvens, dvs. en sekvens, som er komplementær til en i en levende celle forekommende målnukleinsyresekvens, eller en 19-28mer sense-sekvens, som er komplementær til antisense-sekvensen, og - 3'Ni...Ni.i5' er et 3-50mert overhæng af sekvens, der tillader oligomerisation af de dobbeltstrengede oligonukleotider ved intermolekylær vekselvirkning som følge af 3'-overhæng-3'-overhæng-baseparring, eller hvori - 3Ni...Nj5 er en 19-28mer antisense-sekvens, dvs. en sekvens, som er komplementær til en i en levende celle forekommende målnukleinsyresekvens, eller en 19-28mer sense-sekvens, som er komplementær til antisense-sekvensen, og - 3Νι...Νμ5 er et 3-50mert overhæng af sekvens, der tillader oligomerisation af de dobbeltstrengede oligonukleotider ved intermolekylær vekselvirkning under anvendelse af en oligonukleotidlinker, som danner baseparring med oligo-nukleotidernes 3'-overhæng, og hvori - sammensætningen endvidere omfatter oligonukleotidlinkeren.
2. Oligonukleotidsammensætning ifølge krav 1, hvori sekvensen 3 Nj...Nj5 består af 19-21 nukleotider.
3. Oligonukleotidsammensætning ifølge krav 1 eller 2 omfattende sekvenser 3'Νι...Νμ5', der består af 5 til 8 nukleotider.
4. Oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 3, hvori sekvensen ^Ν^,.Νμ-ι5 omfatter stabiliseringsgrupper.
5. Oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 4, hvori sekvensen 3Nj...Nj5' er valgt fra den gruppe, der består af desoxyribonukleotider, ribonukleotider og nukleotidanaloge.
6. Transfektionssammensætning omfattende mindst én oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 5 og et transfektionsmiddel eller -præparat.
7. Transfektionssammensætning ifølge krav 6, hvori transfektionsmidlet er en ikke-viral vektor.
8. Transfektionssammensætning ifølge krav 7, hvori transfektionsmidlet er valgt fra den gruppe, der består af kationiske lipider og kationiske polymere.
9. Fremgangsmåde til fremstilling af en sammensætning af oligonukleotider ifølge et hvilket som helst af kravene 1 til 5 omfattende - syntetisering af oligonukleotidstrenge med sekvenser 3Ν|...Ν,5' og ^...Νμ5' som defineret i et hvilket som helst af kravene 1 til 5 ad en kemisk eller enzymatisk vej; - annealing af de således opnåede syntetiserede oligonukleotidstrenge.
10. Fremgangsmåde ifølge krav 9, som endvidere omfatter tilføjelse af én eller flere linkere efter trinnet til annealing af oligonukleotiderne ifølge et hvilket som helst af kravene 1 til 5, idet linkerne har nukleotidiske sekvensender, der er komplementære til sekvensen 3'Ni...Nm5'.
11. Fremgangsmåde ifølge krav 10, ved hvilken linkeren eller linkerne er valgt fra den gruppe, der består af oligonukleotider, hårnålelignende struktur, kort dobbeltstrenget nukleinsyre med 3'- eller 5-overhæng og dobbeltstrengede oligonukleotider.
12. Fremgangsmåde ifølge krav 11, ved hvilken linkeren er valgt fra den gruppe, der består af desoxyribonukleotider, ribonukleotider og nukleotidanaloge.
13. Fremgangsmåde til inhibering af genekspression in vitro omfattende anvendelse af en oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 5 eller en transfektionssammensætning ifølge et hvilket som helst af kravene 6 til 8.
14. Oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 5 eller transfektionssammensætning ifølge et hvilket som helst af kravene 6 til 8 til anvendelse ved terapi.
15. Oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 5 eller transfektionssammensætning ifølge et hvilket som helst af kravene 6 til 8 til anvendelse ved behandling af cancer eller virusinfektion.
16. Oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 5 eller transfektionssammensætning ifølge et hvilket som helst af kravene 6 til 8 til anvendelse ved behandling af blærecancer, prostatacancer eller leukæmi.
17. Oligonukleotidsammensætning ifølge et hvilket som helst af kravene 1 til 5 eller transfektionssammensætning ifølge et hvilket som helst af kravene 6 til 8 til anvendelse ved behandling af HIV-infektion, hepatitis eller influenza-infektion.
DK06754634.1T 2005-06-01 2006-06-01 Oligonukleotider til RNA-interferens samt biologiske anvendelser deraf DK1888749T3 (da)

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US68601005P 2005-06-01 2005-06-01
PCT/EP2006/006340 WO2006128739A1 (en) 2005-06-01 2006-06-01 Oligonucleotides for rna interference and biological applications thereof

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US (2) US8802640B2 (da)
EP (1) EP1888749B1 (da)
JP (1) JP5371424B2 (da)
CN (1) CN101213300B (da)
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Publication number Priority date Publication date Assignee Title
EP1888749B1 (en) 2005-06-01 2014-10-15 Polyplus Transfection Oligonucleotides for rna interference and biological applications thereof
EP4082551A1 (en) * 2006-08-08 2022-11-02 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5' phosphate oligonucleotides
AU2008242842B2 (en) 2007-04-17 2014-06-05 Baxter Healthcare Sa Nucleic acid microparticles for pulmonary delivery
JP5689413B2 (ja) 2008-05-21 2015-03-25 ライニッシュ フリードリッヒ−ウィルヘルムズ−ユニバーシタット ボン 平滑末端を有する５’三リン酸オリゴヌクレオチドおよびその使用
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
EP3119887B1 (en) * 2014-03-20 2019-02-20 Oommen Varghese Improved small interfering ribonucleic acid molecules
WO2016110691A1 (en) * 2015-01-06 2016-07-14 The University Court Of The University Of Aberdeen Enhanced rnai mediated gene regulation

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US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20070026394A1 (en) * 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US20030212022A1 (en) * 2001-03-23 2003-11-13 Jean-Marie Vogel Compositions and methods for gene therapy
DK1309726T4 (da) 2000-03-30 2019-01-28 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití
EP1888749B1 (en) 2005-06-01 2014-10-15 Polyplus Transfection Oligonucleotides for rna interference and biological applications thereof

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EP1888749A1 (en) 2008-02-20
US20080153772A1 (en) 2008-06-26
US8802640B2 (en) 2014-08-12
US9243248B2 (en) 2016-01-26
US20140343125A1 (en) 2014-11-20
EP1888749B1 (en) 2014-10-15
WO2006128739A1 (en) 2006-12-07
CN101213300B (zh) 2013-01-23
CN101213300A (zh) 2008-07-02
JP2008541745A (ja) 2008-11-27

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