CA2330574A1 - Nucleoside triphosphates et leur integration aux ribozymes - Google Patents

Nucleoside triphosphates et leur integration aux ribozymes Download PDF

Info

Publication number
CA2330574A1
CA2330574A1 CA002330574A CA2330574A CA2330574A1 CA 2330574 A1 CA2330574 A1 CA 2330574A1 CA 002330574 A CA002330574 A CA 002330574A CA 2330574 A CA2330574 A CA 2330574A CA 2330574 A1 CA2330574 A1 CA 2330574A1
Authority
CA
Canada
Prior art keywords
nucleic acid
rna
acid molecule
enzymatic nucleic
rna polymerase
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
CA002330574A
Other languages
English (en)
Inventor
Leonid Beigelman
Alex Burgin
Amber Beaudry
Alexander Karpeisky
Jasenka Matulic-Adamic
David Sweedler
Shawn Zinnen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirna Therapeutics Inc
Original Assignee
Individual
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US09/186,675 external-priority patent/US6127535A/en
Application filed by Individual filed Critical Individual
Publication of CA2330574A1 publication Critical patent/CA2330574A1/fr
Abandoned legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals
    • C07H19/10Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16Purine radicals
    • C07H19/20Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/345Spatial arrangement of the modifications having at least two different backbone modifications

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

L'invention concerne de nouveaux nucléoside triphosphates, des procédés de synthèse et une procédure d'intégration de ces nucléoside triphosphates aux oligonucléotides ainsi que l'isolement de nouveaux catalyseurs d'acides nucléiques (p.ex., de ribozymes).
CA002330574A 1998-04-29 1999-04-28 Nucleoside triphosphates et leur integration aux ribozymes Abandoned CA2330574A1 (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US8372798P 1998-04-29 1998-04-29
US60/083,727 1998-04-29
US09/186,675 US6127535A (en) 1997-11-05 1998-11-04 Nucleoside triphosphates and their incorporation into oligonucleotides
US09/186,675 1998-11-04
PCT/US1999/009348 WO1999055857A2 (fr) 1998-04-29 1999-04-28 Nucleoside triphosphates et leur integration aux ribozymes

Publications (1)

Publication Number Publication Date
CA2330574A1 true CA2330574A1 (fr) 1999-11-04

Family

ID=26769652

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002330574A Abandoned CA2330574A1 (fr) 1998-04-29 1999-04-28 Nucleoside triphosphates et leur integration aux ribozymes

Country Status (5)

Country Link
EP (1) EP1073732A2 (fr)
JP (2) JP2002512794A (fr)
AU (1) AU751480B2 (fr)
CA (1) CA2330574A1 (fr)
WO (1) WO1999055857A2 (fr)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
AU3751299A (en) 1998-04-20 1999-11-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
WO2000075306A2 (fr) 1999-04-30 2000-12-14 Cyclops Genome Sciences Limited Polynucléotides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
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
CA2397813A1 (fr) * 2000-02-24 2001-08-30 Ira Von Carlowitz Methode et reactif d'inhibition de grid
WO2002011674A2 (fr) * 2000-08-09 2002-02-14 Ribozyme Pharmaceuticals, Incorporated Procede et reactif permettant d'inhiber le canal chlorure active par le calcium 1 (clca-1)
DE60144479D1 (fr) 2000-09-01 2011-06-01 Ribozyme Pharm Inc
WO2002044425A2 (fr) 2000-12-01 2002-06-06 Visigen Biotechnologies, Inc. Synthese d'acides nucleiques d'enzymes, et compositions et methodes modifiant la fidelite d'incorporation de monomeres
EP1572067A4 (fr) 2001-05-18 2009-05-13 Sirna Therapeutics Inc Conjugues et compositions pour administration cellulaire
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US7071311B2 (en) 2002-02-13 2006-07-04 Sirna Therapeutics, Inc. Antibodies having specificity for 2′-C-allyl nucleic acids
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
GB2413557B (en) * 2002-02-20 2006-08-16 Sirna Therapeutics Inc RNA interference mediated inhibtion of gene expression using chemically modified short interfering nucleic acid (siNA)
GB2406568B (en) * 2002-02-20 2005-09-28 Sirna Therapeutics Inc RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US6989442B2 (en) 2002-07-12 2006-01-24 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US7655790B2 (en) 2002-07-12 2010-02-02 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
AU2012216354B2 (en) * 2002-08-05 2016-01-14 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
SI3222724T1 (sl) 2002-08-05 2019-03-29 Silence Therapeutics Gmbh Nadaljnje nove oblike molekul interferenčne RNA
WO2004083432A1 (fr) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire
EP3222294A1 (fr) 2003-04-30 2017-09-27 Sirna Therapeutics, Inc. Conjugués et compositions pour délivrer des composés au niveau cellulaire
CA2554212A1 (fr) 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel)
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
WO2006042112A2 (fr) * 2004-10-05 2006-04-20 California Institute Of Technology Acides nucleiques a regulation d'aptameres et leurs utilisations
EP4082551A1 (fr) 2006-08-08 2022-11-02 Rheinische Friedrich-Wilhelms-Universität Bonn Structure et utilisation d'oligonucléotides 5'-phosphate
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
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
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
JP6106085B2 (ja) 2010-08-24 2017-03-29 サーナ・セラピューティクス・インコーポレイテッドSirna Therapeutics,Inc. 内部非核酸スペーサーを含む一本鎖RNAi剤
CN103209987B (zh) 2010-09-22 2017-06-06 艾丽奥斯生物制药有限公司 取代的核苷酸类似物
DK2632472T3 (en) 2010-10-29 2018-03-19 Sirna Therapeutics Inc RNA INTERFERENCE-MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERRING NUCLEIC ACIDS (SINA)
EP2508530A1 (fr) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification d'oligonucléotides triphosphorylés au moyen d'étiquettes de capture
AU2012358804B2 (en) 2011-12-22 2018-04-19 Alios Biopharma, Inc. Substituted phosphorothioate nucleotide analogs
EP4043039A1 (fr) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Oligonucléotides de modulation arn ayant des caractéristiques améliorées pour le traitement de la dystrophie musculaire de duchenne et becker
WO2013142124A1 (fr) 2012-03-21 2013-09-26 Vertex Pharmaceuticals Incorporated Formes solides d'un promédicament nucléotidique thiophosphoramidate
US9012427B2 (en) 2012-03-22 2015-04-21 Alios Biopharma, Inc. Pharmaceutical combinations comprising a thionucleotide analog
US9115160B2 (en) * 2012-08-31 2015-08-25 Sunny Pharmtech Inc. Solvent-free process for the preparation of cyclophosphamide
EP2712870A1 (fr) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Nouveaux ligands de RIG-I et procédés pour les produire

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU1775405C (ru) * 1987-10-29 1992-11-15 Институт цитологии и генетики СО АН СССР Способ получени 2 @ -дезоксинуклеозид-5 @ -0-(1-тио)трифосфатов
US4879214A (en) * 1988-11-15 1989-11-07 E. I. Du Pont De Nemours And Company Differentiation of nucleic acid segments on the basis of nucleotide differences
US5610054A (en) * 1992-05-14 1997-03-11 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecule targeted against Hepatitis C virus
DE69415343T2 (de) * 1993-10-27 1999-08-26 Ribozyme Pharmaceuticals 2'-amido-und 2'-peptido-modifizierte oligonukleotide
EP0767657A4 (fr) * 1994-06-22 1999-01-20 Nexstar Pharmaceuticals Inc Nouveau procede de production de nucleosides connus et nouveaux, modifies en position 2' par deplacement nucleophile intramoleculaire
AU4412096A (en) * 1994-12-13 1996-07-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
WO1997026270A2 (fr) * 1996-01-16 1997-07-24 Ribozyme Pharmaceuticals, Inc. Synthese de methoxy nucleosides et de molecules d'acide nucleique enzymatique
JP2001525667A (ja) * 1997-05-09 2001-12-11 リボザイム・ファーマシューティカルズ・インコーポレーテッド C−rafの発現レベルに関係した疾患または状態の酵素核酸処置

Also Published As

Publication number Publication date
WO1999055857A3 (fr) 2000-02-24
JP2002512794A (ja) 2002-05-08
EP1073732A2 (fr) 2001-02-07
WO1999055857A2 (fr) 1999-11-04
JP2004147666A (ja) 2004-05-27
AU3872499A (en) 1999-11-16
AU751480B2 (en) 2002-08-15

Similar Documents

Publication Publication Date Title
AU751480B2 (en) Nucleoside triphosphates and their incorporation into ribozymes
US6528640B1 (en) Synthetic ribonucleic acids with RNAse activity
US6617438B1 (en) Oligoribonucleotides with enzymatic activity
US6093555A (en) Amido and peptido modified enzymatic nucleic acid molecules
US6365374B1 (en) 2′-deoxy-2′-alkylnucleotide containing nucleic acid
US6831171B2 (en) Nucleic acid catalysts with endonuclease activity
US6110462A (en) Enzymatic DNA molecules that contain modified nucleotides
US20030004122A1 (en) Nucleotide triphosphates and their incorporation into oligonucleotides
US6506888B1 (en) 2′-O-amino-containing nucleoside analogs and polynucleotides
US6605713B1 (en) Mirror-symmetrical selection and evolution of nucleic acids
US5627053A (en) 2'deoxy-2'-alkylnucleotide containing nucleic acid
JP2003525017A (ja) 遺伝子発現を調節しうる新規な化学組成を有する核酸分子
JP3903392B2 (ja) ヌクレオシド類似体の化学合成とそのポリヌクレオチドへの導入
EP1212416A2 (fr) Modulateurs d'expression genetique a base d'acide nucleique
US6482932B1 (en) Nucleoside triphosphates and their incorporation into oligonucleotides
WO1998043993A2 (fr) Acides nucleiques en tant que catalyseurs
US20030125291A1 (en) Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
US6127535A (en) Nucleoside triphosphates and their incorporation into oligonucleotides
EP1493818A2 (fr) Nucléosides triphosphates et leur intégration aux ribozymes
AU2004200709A1 (en) Nucleoside Triphosphates and their Incorporation into Ribozymes
CA2334161A1 (fr) Utilisation de molecules d'acide nucleique comme agents antiviraux
US20030105308A1 (en) Nucleoside triphosphates and their incorporation into oligonucleotides
US20040102413A1 (en) Method and reagent for the inhibition of telomerase enzyme
WO1999020753A2 (fr) Formation de liaison peptidique utilisant des catalyseurs d'acide nucleique
AU2003264633A1 (en) Nucleic Acid Molecules with Novel Chemical Compositions Capable of Modulating Gene Expression

Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued