WO2004063375A1 - Optimisation d'arnsi par arni antisens - Google Patents
Optimisation d'arnsi par arni antisens Download PDFInfo
- Publication number
- WO2004063375A1 WO2004063375A1 PCT/NO2004/000007 NO2004000007W WO2004063375A1 WO 2004063375 A1 WO2004063375 A1 WO 2004063375A1 NO 2004000007 W NO2004000007 W NO 2004000007W WO 2004063375 A1 WO2004063375 A1 WO 2004063375A1
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- WO
- WIPO (PCT)
- Prior art keywords
- sirna
- rnai antisense
- molecules
- rnai
- nucleic acid
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
Definitions
- RNA sequence-specific mediators of RNA interference regards RNA sequence-specific mediators of RNA interference and relates to isolated RNA molecules (double stranded; single stranded) of from about 21 -23 nucleotides in general.
- WO 01/75164 also relates inter alia to a method of producing said RNA molecules, e.g. by using the Drosophila in vitro system, by chemical synthesis or recombinant techniques.
- WO 02/44321 disclose inter alia isolated double-stranded RNA molecule of 19-25 nucleotides capable of target-specific nucleic acid modifications, a method for processing said RNA molecules and the use thereof.
- siRNA modifications would be affected by siRNA modifications, chemical or mutational, in different ways, and generally in a deleterious way, as shown in the exemplary material. Therefore, there is a considerable need for a method to efficiently identify optimal siRNA molecules, which may be chemical modified or not, to be able to develop useful pharmaceutical agents to modulate the expression of a target gene.
- EP 0 756 634 Bl disclose a method for the screening of a genetic sequence which is capable of inhibiting, reducing, altering or otherwise modulate the expression of a target nucleotide sequence, e.g. the screening for useful antisense, sense or ribozyme constructs or other nucleic sequences. More specifically, the method disclosed in EP 0 756 634 Bl makes use of & pombe to evaluate the effect of the introduction of the molecule to be tested on the expression of a target gene in S. pombe.
- alkylation such as methylation
- 3' or 5' end modifications such as fluorescent labels, non-standard nucleotides, lipophilic linker molecules or peptides
- modification or exchange of the phosphodiester bond e.g. with phosporothioates, methylphosphonates, or polyamide.
- RNAi antisense could also be expressed endogenouslyfrom appropriate vectors, preferably of viral origin and from RNA polymerase III promoters.
- appropriate vectors preferably of viral origin and from RNA polymerase III promoters.
- primer extension analysis which identifies the most prominent cleavage positions within the target mRNA. From the known sequence of target mRNA, the sequence of RNAi antisense causing the most prominent cleavage events can be inferred.
- RNAi antisense has target position dependence
- a comparison between RNAi antisense and siRNA can, of course, not conclusively prove a shared RNAi pathway, but major deviations between the two could very well decisively falsify our hypothesis of a shared RNAi pathway.
- the efficacies of antisense RNAs targeting sites on TF mRNA coding region from the start-codon (as-77) to the stop- codon (as-929) were evaluated in our quantitative Northern assay (Fig. IB).
- RNAi antisense reaches maximum activity faster than siRNA
- RNA interference is mediated by 21 and 22 nt RNAs. Genes Dev., 15, 188-200.
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La présente invention concerne un nouveau procédé d'identification de séquences efficaces d'ARNsi pouvant être utilisées pour modifier l'expression d'une séquence de gènes cible. De manière spécifique, cette invention concerne un procédé d'identification de sites de ciblage d'ARNsi optimal à l'aide d'ARNi antisens. En outre, ladite invention concerne des molécules d'ARNsi identifiées par le présent procédé de criblage ainsi que des préparations pharmaceutiques contenant lesdites molécules d'ARNsi.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US44003603P | 2003-01-15 | 2003-01-15 | |
US60/440,036 | 2003-01-15 | ||
NO20030206 | 2003-01-15 | ||
NO20030206A NO20030206D0 (no) | 2003-01-15 | 2003-01-15 | Screening metode |
Publications (1)
Publication Number | Publication Date |
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WO2004063375A1 true WO2004063375A1 (fr) | 2004-07-29 |
Family
ID=32716487
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/NO2004/000007 WO2004063375A1 (fr) | 2003-01-15 | 2004-01-14 | Optimisation d'arnsi par arni antisens |
Country Status (1)
Country | Link |
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WO (1) | WO2004063375A1 (fr) |
Cited By (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US7459547B2 (en) | 2003-06-02 | 2008-12-02 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
US8304530B2 (en) | 2003-06-02 | 2012-11-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
US8309704B2 (en) | 2003-06-02 | 2012-11-13 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNAi |
JP2012223202A (ja) * | 2004-12-22 | 2012-11-15 | Alnylam Pharmaceuticals Inc | 遺伝子サイレンシングに有用なhbvおよびhcv保存配列 |
US8329463B2 (en) | 2000-12-01 | 2012-12-11 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8394628B2 (en) | 2000-03-30 | 2013-03-12 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US8729036B2 (en) | 2002-08-07 | 2014-05-20 | University Of Massachusetts | Compositions for RNA interference and methods of use thereof |
US9879253B2 (en) | 2003-12-22 | 2018-01-30 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
US10392619B2 (en) | 2009-10-12 | 2019-08-27 | Larry J. Smith | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
US10513703B2 (en) | 2014-11-10 | 2019-12-24 | Alnylam Pharmaceuticals, Inc. | Hepatitis B virus (HBV) iRNA compositions and methods of use thereof |
US11324820B2 (en) | 2017-04-18 | 2022-05-10 | Alnylam Pharmaceuticals, Inc. | Methods for the treatment of subjects having a hepatitis b virus (HBV) infection |
US11492623B2 (en) | 2018-08-13 | 2022-11-08 | Alnylam Pharmaceuticals, Inc. | Hepatitis B virus (HBV) dsRNA agent compositions and methods of use thereof |
US11926817B2 (en) | 2019-08-09 | 2024-03-12 | Nutcracker Therapeutics, Inc. | Microfluidic apparatus and methods of use thereof |
Citations (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5525468A (en) * | 1992-05-14 | 1996-06-11 | Ribozyme Pharmaceuticals, Inc. | Assay for Ribozyme target site |
WO2002090590A1 (fr) * | 1992-09-29 | 2002-11-14 | Isis Pharmaceuticals, Inc. | Modulation antisens de l'expression de sous unites de l'interleukine 12 p35 |
WO2003100093A2 (fr) * | 2002-05-28 | 2003-12-04 | Isis Innovation Ltd. | Procede de selection de cibles pour silençage genique par interference d'arn |
-
2004
- 2004-01-14 WO PCT/NO2004/000007 patent/WO2004063375A1/fr active Application Filing
Patent Citations (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5525468A (en) * | 1992-05-14 | 1996-06-11 | Ribozyme Pharmaceuticals, Inc. | Assay for Ribozyme target site |
WO2002090590A1 (fr) * | 1992-09-29 | 2002-11-14 | Isis Pharmaceuticals, Inc. | Modulation antisens de l'expression de sous unites de l'interleukine 12 p35 |
WO2003100093A2 (fr) * | 2002-05-28 | 2003-12-04 | Isis Innovation Ltd. | Procede de selection de cibles pour silençage genique par interference d'arn |
Non-Patent Citations (7)
Title |
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AMARZGUIOUI M ET AL: "Tolerance for mutations and chemical modifications in a siRNA", NUCLEIC ACIDS RESEARCH, vol. 31, no. 2, 15 January 2003 (2003-01-15), pages 589 - 595, XP002270887, ISSN: 0305-1048 * |
ELBASHIR SAYDA M ET AL: "RNA interference is mediated by 21- and 22-nucleotide RNAs", GENES AND DEVELOPMENT, vol. 15, no. 2, 15 January 2001 (2001-01-15), pages 188 - 200, XP002204651, ISSN: 0890-9369 * |
HOLEN T ET AL: "Positional effects of short interfering RNAs targeting the human coagulation trigger Tissue Factor", NUCLEIC ACIDS RESEARCH, vol. 30, no. 8, 15 April 2002 (2002-04-15), pages 1757 - 1766, XP002232890, ISSN: 0305-1048 * |
HOLEN TORGEIR ET AL: "Similar behaviour of single-strand and double-strand siRNAs suggests they act through a common RNAi pathway.", NUCLEIC ACIDS RESEARCH, vol. 31, no. 9, 1 May 2003 (2003-05-01), pages 2401 - 2407, XP002283906, ISSN: 0305-1048 * |
SCHWARZ D S ET AL: "EVIDENCE THAT SIRNAS FUNCTION AS GUIDES, NOT PRIMERS, IN THE DROSOPHILA AND HUMAN RNAI PATHWAYS", MOLECULAR CELL, vol. 10, no. 3, September 2002 (2002-09-01), pages 537 - 548, XP009019083, ISSN: 1097-2765 * |
SOHAIL M ET AL: "OLIGONUCLEOTIDE SCANNING ARRAYS: APPLICATION TO HIGH-THROUGHPUT SCREENING FOR EFFECTIVE ANTISENSE REAGENTS AND THE STUDY OF NUCLEIC ACID INTERACTIONS", ADVANCES IN BIOCHEMICAL ENGINEERING, BIOTECHNOLOGY, SPRINGER, BERLIN, DE, vol. 77, 2002, pages 43 - 56, XP008026711, ISSN: 0724-6145 * |
VICKERS TIMOTHY A ET AL: "Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents: A comparative analysis.", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 278, no. 9, 28 February 2003 (2003-02-28), pages 7108 - 7118, XP002283905, ISSN: 0021-9258 * |
Cited By (47)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8394628B2 (en) | 2000-03-30 | 2013-03-12 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US10472625B2 (en) | 2000-03-30 | 2019-11-12 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA sequence-specific mediators of RNA interference |
US9193753B2 (en) | 2000-03-30 | 2015-11-24 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US9012138B2 (en) | 2000-03-30 | 2015-04-21 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA sequence-specific mediators of RNA interference |
US9012621B2 (en) | 2000-03-30 | 2015-04-21 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA sequence-specific mediators of RNA interference |
US8790922B2 (en) | 2000-03-30 | 2014-07-29 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA sequence-specific mediators of RNA interference |
US8742092B2 (en) | 2000-03-30 | 2014-06-03 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US8632997B2 (en) | 2000-03-30 | 2014-01-21 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US8552171B2 (en) | 2000-03-30 | 2013-10-08 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US8420391B2 (en) | 2000-03-30 | 2013-04-16 | University Of Massachusetts | RNA sequence-specific mediators of RNA interference |
US8853384B2 (en) | 2000-12-01 | 2014-10-07 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8372968B2 (en) | 2000-12-01 | 2013-02-12 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8362231B2 (en) | 2000-12-01 | 2013-01-29 | Max-Planck-Gesellschaft zur Föderung der Wissenschaften E.V. | RNA interference mediating small RNA molecules |
US8445237B2 (en) | 2000-12-01 | 2013-05-21 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8329463B2 (en) | 2000-12-01 | 2012-12-11 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US10633656B2 (en) | 2000-12-01 | 2020-04-28 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | RNA interference mediating small RNA molecules |
US8895721B2 (en) | 2000-12-01 | 2014-11-25 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8895718B2 (en) | 2000-12-01 | 2014-11-25 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8765930B2 (en) | 2000-12-01 | 2014-07-01 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8778902B2 (en) | 2000-12-01 | 2014-07-15 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8933044B2 (en) | 2000-12-01 | 2015-01-13 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8796016B2 (en) | 2000-12-01 | 2014-08-05 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US8993745B2 (en) | 2000-12-01 | 2015-03-31 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA interference mediating small RNA molecules |
US9611472B2 (en) | 2002-08-07 | 2017-04-04 | University Of Massachusetts | Compositions for RNA interference and methods of use thereof |
US8729036B2 (en) | 2002-08-07 | 2014-05-20 | University Of Massachusetts | Compositions for RNA interference and methods of use thereof |
US8304530B2 (en) | 2003-06-02 | 2012-11-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
US7732593B2 (en) | 2003-06-02 | 2010-06-08 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
US7459547B2 (en) | 2003-06-02 | 2008-12-02 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
US8309704B2 (en) | 2003-06-02 | 2012-11-13 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNAi |
US8309705B2 (en) | 2003-06-02 | 2012-11-13 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
US9121018B2 (en) | 2003-06-02 | 2015-09-01 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
US7772203B2 (en) | 2003-06-02 | 2010-08-10 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
US10604754B2 (en) | 2003-06-02 | 2020-03-31 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
US11459562B2 (en) | 2003-06-02 | 2022-10-04 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
US10364429B2 (en) | 2003-06-02 | 2019-07-30 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
US8329892B2 (en) | 2003-06-02 | 2012-12-11 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
US10385339B2 (en) | 2003-12-22 | 2019-08-20 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
US9879253B2 (en) | 2003-12-22 | 2018-01-30 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
JP2015003913A (ja) * | 2004-12-22 | 2015-01-08 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | 遺伝子サイレンシングに有用なhbvおよびhcv保存配列 |
JP2012223202A (ja) * | 2004-12-22 | 2012-11-15 | Alnylam Pharmaceuticals Inc | 遺伝子サイレンシングに有用なhbvおよびhcv保存配列 |
US10392619B2 (en) | 2009-10-12 | 2019-08-27 | Larry J. Smith | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
US11359201B2 (en) | 2009-10-12 | 2022-06-14 | Larry J. Smith | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
US11060091B2 (en) | 2014-11-10 | 2021-07-13 | Alnylam Pharmaceuticals, Inc. | Hepatitis B virus (HBV) iRNA compositions and methods of use thereof |
US10513703B2 (en) | 2014-11-10 | 2019-12-24 | Alnylam Pharmaceuticals, Inc. | Hepatitis B virus (HBV) iRNA compositions and methods of use thereof |
US11324820B2 (en) | 2017-04-18 | 2022-05-10 | Alnylam Pharmaceuticals, Inc. | Methods for the treatment of subjects having a hepatitis b virus (HBV) infection |
US11492623B2 (en) | 2018-08-13 | 2022-11-08 | Alnylam Pharmaceuticals, Inc. | Hepatitis B virus (HBV) dsRNA agent compositions and methods of use thereof |
US11926817B2 (en) | 2019-08-09 | 2024-03-12 | Nutcracker Therapeutics, Inc. | Microfluidic apparatus and methods of use thereof |
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