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

Nucleoside triphosphates et leur integration aux ribozymes Download PDF

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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

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Classifications

    • 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
    • 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
    • C12N15/113—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
    • C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications

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 1998-11-04
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)
WO1999054459A2 (fr) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Molecules d'acides nucleiques presentant de nouvelles compositions chimiques capables de moduler l'expression genique
WO2000066605A2 (fr) * 1999-04-30 2000-11-09 Cyclops Genome Sciences Limited Polynucleotides
WO2005041859A2 (fr) 2003-04-30 2005-05-12 Sirna Therapeutics, Inc. Conjugues et compositions
WO2002081628A2 (fr) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation de l'expression genique associee a la proliferation inflammatoire et a la croissance de neurites, par des procedes faisant intervenir l'acide nucleique
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
CA2397813A1 (fr) * 2000-02-24 2001-08-30 Ira Von Carlowitz Methode et reactif d'inhibition de grid
AU2001283222A1 (en) * 2000-08-09 2002-02-18 Ribozyme Pharmaceuticals, Incorporated Method and reagent for the inhibition of calcium activated chloride channel-1 (clca-1)
CA2421040C (fr) 2000-09-01 2010-01-12 Ribozyme Pharmaceuticals, Incorporated Procede de synthese de nucleosides, de nucleosides en derivant, et de leurs derives non nucleosides
EP1354064A2 (fr) 2000-12-01 2003-10-22 Visigen Biotechnologies, Inc. Synthese d'acides nucleiques d'enzymes, et compositions et methodes modifiant la fidelite d'incorporation de monomeres
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
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)
EP3231445A1 (fr) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Conjugués et compositions pour la fourniture cellulaire
US7071311B2 (en) 2002-02-13 2006-07-04 Sirna Therapeutics, Inc. Antibodies having specificity for 2′-C-allyl nucleic acids
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)
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)
US7655790B2 (en) 2002-07-12 2010-02-02 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US6989442B2 (en) 2002-07-12 2006-01-24 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
EP1857547B2 (fr) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Autres nouvelles formes d'ARN interférants
AU2012216354B2 (en) * 2002-08-05 2016-01-14 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
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
EP1713915B1 (fr) 2004-02-10 2009-12-16 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
EP2056845B1 (fr) 2006-08-08 2017-10-11 Rheinische Friedrich-Wilhelms-Universität Bonn Structure et utilisation d'oligonucléotides 5'-phosphate
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
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
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
WO2009141146A1 (fr) 2008-05-21 2009-11-26 Gunther Hartmann Oligonucléotide à 5’-triphosphate présentant une extrémité franche et ses utilisations
NZ595955A (en) 2009-04-24 2012-10-26 Prosensa Technologies Bv Oligonucleotide comprising an inosine for treating dmd
WO2012027206A1 (fr) 2010-08-24 2012-03-01 Merck Sharp & Dohme Corp. Agents à base d'arni à un seul brin contenant une séquence intercalaire interne ne correspondant pas à un acide nucléique
US8871737B2 (en) 2010-09-22 2014-10-28 Alios Biopharma, Inc. Substituted nucleotide analogs
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering 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
WO2013096680A1 (fr) 2011-12-22 2013-06-27 Alios Biopharma, Inc. Analogues de nucléotide phosphorothioate substitués
CN104203289B (zh) 2012-01-27 2020-11-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
EP2828277A1 (fr) 2012-03-21 2015-01-28 Vertex Pharmaceuticals Incorporated Formes solides d'un promédicament nucléotidique thiophosphoramidate
WO2013142157A1 (fr) 2012-03-22 2013-09-26 Alios Biopharma, Inc. Combinaisons pharmaceutiques comprenant un analogue thionucléotidique
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
RU1775406C (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
JPH09504297A (ja) * 1993-10-27 1997-04-28 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 ２′−アミドおよび２′−ペプチド修飾オリゴヌクレオチド
AU710074B2 (en) * 1994-06-22 1999-09-16 Proligo Llc Novel method of preparation of known and novel 2'-modified nucleosides by intramolecular nucleophilic displacement
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
AU1430097A (en) * 1996-01-16 1997-08-11 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
CA2288640A1 (fr) * 1997-05-09 1998-11-12 Ribozyme Pharmaceuticals, Inc. Traitement a l'acide nucleique enzymatique de maladies et etats pathologiques associes aux niveaux d'expression de c-raf

Also Published As

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

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Legal Events

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EEER Examination request
FZDE Discontinued