WO1998043993A3 - Nucleic acid catalysts - Google Patents

Nucleic acid catalysts Download PDF

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Publication number
WO1998043993A3
WO1998043993A3 PCT/US1998/006231 US9806231W WO9843993A3 WO 1998043993 A3 WO1998043993 A3 WO 1998043993A3 US 9806231 W US9806231 W US 9806231W WO 9843993 A3 WO9843993 A3 WO 9843993A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
acid catalysts
synthesis
methods
acid molecules
Prior art date
Application number
PCT/US1998/006231
Other languages
French (fr)
Other versions
WO1998043993A2 (en
Inventor
Ronald R Breaker
Original Assignee
Univ Yale
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
Application filed by Univ Yale filed Critical Univ Yale
Priority to AU65917/98A priority Critical patent/AU6591798A/en
Publication of WO1998043993A2 publication Critical patent/WO1998043993A2/en
Publication of WO1998043993A3 publication Critical patent/WO1998043993A3/en

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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/121—Hammerhead

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Catalysts (AREA)

Abstract

Nucleic acid molecules with new motifs having catalytic activity, methods of synthesis, and use thereof are also described.
PCT/US1998/006231 1997-03-31 1998-03-30 Nucleic acid catalysts WO1998043993A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU65917/98A AU6591798A (en) 1997-03-31 1998-03-30 Nucleic acid catalysts

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US4290597P 1997-03-31 1997-03-31
US60/042,905 1997-03-31

Publications (2)

Publication Number Publication Date
WO1998043993A2 WO1998043993A2 (en) 1998-10-08
WO1998043993A3 true WO1998043993A3 (en) 1999-04-15

Family

ID=21924366

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/006231 WO1998043993A2 (en) 1997-03-31 1998-03-30 Nucleic acid catalysts

Country Status (2)

Country Link
AU (1) AU6591798A (en)
WO (1) WO1998043993A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9499818B2 (en) 2007-10-26 2016-11-22 BioMarin Technologies, B.V. Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002514913A (en) * 1996-12-19 2002-05-21 エール ユニバーシティ Bioreactive allosteric polynucleotide
AU6522599A (en) * 1998-10-23 2000-05-15 Children's Medical Center Corporation Use of a self-cleaving rna motif to modulate gene expression
EP1092777A1 (en) * 1999-10-15 2001-04-18 Aventis Pharma S.A. Self-cleaving RNA sequences and their use for the control of protein synthesis.
US6831171B2 (en) 2000-02-08 2004-12-14 Yale University Nucleic acid catalysts with endonuclease activity
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
AU2002316135B9 (en) 2001-05-18 2009-05-28 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
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
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)
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)
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP1622572B1 (en) 2003-04-30 2017-12-20 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20060200878A1 (en) 2004-12-21 2006-09-07 Linda Lutfiyya Recombinant DNA constructs and methods for controlling gene expression
EP3372676A1 (en) * 2004-12-21 2018-09-12 Monsanto Technology, LLC Recombinant dna constructs and methods for controlling gene expression
EP3199646A1 (en) 2006-10-12 2017-08-02 Monsanto Technology LLC Plant micrornas and methods of use thereof
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
AU2010239779A1 (en) 2009-04-24 2011-11-17 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating DMD
DK2632472T3 (en) 2010-10-29 2018-03-19 Sirna Therapeutics Inc RNA INTERFERENCE-MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERRING NUCLEIC ACIDS (SINA)
WO2013112053A1 (en) 2012-01-27 2013-08-01 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995019433A2 (en) * 1994-01-12 1995-07-20 Targetech, Inc. Anti-hepatitis b poly- and oligonucleotides
WO1996003152A1 (en) * 1994-07-28 1996-02-08 Georgetown University Antisense oligonucleotides against hepatitis b viral replication
WO1997010328A2 (en) * 1995-07-13 1997-03-20 Ribozyme Pharmaceuticals, Inc. Compositions and method for modulation of gene expression in plants

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995019433A2 (en) * 1994-01-12 1995-07-20 Targetech, Inc. Anti-hepatitis b poly- and oligonucleotides
WO1996003152A1 (en) * 1994-07-28 1996-02-08 Georgetown University Antisense oligonucleotides against hepatitis b viral replication
WO1997010328A2 (en) * 1995-07-13 1997-03-20 Ribozyme Pharmaceuticals, Inc. Compositions and method for modulation of gene expression in plants

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BREAKER, R.R. & JOYCE, G.F.: "Inventing and improving ribozyme function: ...", TIBTECH, vol. 12, 1994, pages 268 - 275, XP002084353 *
NISHIKAWA, F. ET AL.: "Selection in vitro of trans-acting genomic hepatitis delta virus (HDV) ribozymes", EUR. J. BIOCHEM., vol. 237, 1996, pages 712 - 718, XP002084352 *
PERROTTA, A.T. & BEEN, M.D.: "Core sequences and a cleavage site wobble pair required for HDV antigenomic ribozyme self-cleavage", NUCLEIC ACIDS RESEARCH, vol. 24, no. 7, 1996, pages 1314 - 1321, XP000582452 *
TANG, J. & BREAKER, R.R.: "Examination of the catalytic fitness of the hammerhead ribozyme by in vitro selection", RNA, vol. 3, 1997, pages 914 - 925, XP002084354 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9499818B2 (en) 2007-10-26 2016-11-22 BioMarin Technologies, B.V. Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA

Also Published As

Publication number Publication date
WO1998043993A2 (en) 1998-10-08
AU6591798A (en) 1998-10-22

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