WO1993022434A3 - Targeted cleavage of rna using eukaryotic ribonuclease p and external guide sequence - Google Patents

Targeted cleavage of rna using eukaryotic ribonuclease p and external guide sequence Download PDF

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Publication number
WO1993022434A3
WO1993022434A3 PCT/US1993/003961 US9303961W WO9322434A3 WO 1993022434 A3 WO1993022434 A3 WO 1993022434A3 US 9303961 W US9303961 W US 9303961W WO 9322434 A3 WO9322434 A3 WO 9322434A3
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WO
WIPO (PCT)
Prior art keywords
egs
rna
rnaase
cleavage
loop
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.)
Ceased
Application number
PCT/US1993/003961
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French (fr)
Other versions
WO1993022434A2 (en
Inventor
Yan Yuan
Cecilia L Guerrier-Takada
Sidney Altman
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.)
Yale University
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Yale University
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 Yale University filed Critical Yale University
Priority to DE69303712T priority Critical patent/DE69303712T2/en
Priority to AU41198/93A priority patent/AU669367B2/en
Priority to EP93910848A priority patent/EP0638121B1/en
Priority to JP5519468A priority patent/JP3015463B2/en
Publication of WO1993022434A2 publication Critical patent/WO1993022434A2/en
Publication of WO1993022434A3 publication Critical patent/WO1993022434A3/en
Anticipated expiration legal-status Critical
Priority to GR960402216T priority patent/GR3020859T3/en
Ceased legal-status Critical Current

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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
    • 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
    • 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
    • C12N2310/126—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes involving RNAse P

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  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

It has been discovered that any RNA can be targeted for cleavage by RNAase P from eukaryotic cells, for example, human HeLa cells, using a suitably designed oligoribonucleotide ('external guide sequence', or EGS) to form a hybrid with the target RNA, thereby creating a substrate for cleavage by RNAase P in vitro. There are two classes of EGS that can target RNA for cleavage by human RNAase P. These are prepared by transcription from a small DNA fragment that contains a sequence coding for an RNA that can assume a tRNA-like secondary structure. In the first class, the structure lacks at least the first thirteen nucleotides from the 5' terminus of the tRNA-like sequence, the anticodon stem and loop, the variable loop or part of the variable loop. The most efficient EGS with human RNAase P is the EGS in which the anticodon stem and loop was deleted. In the second class, the EGS has changes in both the equivalent of the T-loop and the anticodon stem of the tRNA-like segment of the EGS. Methods are also disclosed to randomly select and to express a suitable EGS in vivo to make a selected RNA a target for cleavage by the host cell RNAase P, thus preventing expression of the function of the target RNA. The methods and compositions should be useful to prevent the expression of disease-causing genes in vivo.
PCT/US1993/003961 1992-04-28 1993-04-28 Targeted cleavage of rna using eukaryotic ribonuclease p and external guide sequence Ceased WO1993022434A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
DE69303712T DE69303712T2 (en) 1992-04-28 1993-04-28 TARGETED CUTTING OF RNA BY MEANS OF EUKARYONTIC RNASE P AND EXTERNAL LEADING SEQUENCE
AU41198/93A AU669367B2 (en) 1992-04-28 1993-04-28 Targeted cleavage of RNA using eukaryotic ribonuclease P and external guide sequence
EP93910848A EP0638121B1 (en) 1992-04-28 1993-04-28 Targeted cleavage of rna using eukaryotic ribonuclease p and external guide sequence
JP5519468A JP3015463B2 (en) 1992-04-28 1993-04-28 Targeted cleavage of RNA using eukaryotic ribonuclease P and external guide sequences
GR960402216T GR3020859T3 (en) 1992-04-28 1996-08-22 Targeted cleavage of rna using eukaryotic ribonuclease p and external guide sequence.

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US87509992A 1992-04-28 1992-04-28
US875,099 1992-04-28
US93193792A 1992-08-18 1992-08-18
US931,937 1992-08-18

Publications (2)

Publication Number Publication Date
WO1993022434A2 WO1993022434A2 (en) 1993-11-11
WO1993022434A3 true WO1993022434A3 (en) 1993-12-23

Family

ID=27128368

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1993/003961 Ceased WO1993022434A2 (en) 1992-04-28 1993-04-28 Targeted cleavage of rna using eukaryotic ribonuclease p and external guide sequence

Country Status (10)

Country Link
EP (1) EP0638121B1 (en)
JP (1) JP3015463B2 (en)
AT (1) ATE140482T1 (en)
AU (1) AU669367B2 (en)
CA (1) CA2117903C (en)
DE (1) DE69303712T2 (en)
DK (1) DK0638121T3 (en)
ES (1) ES2093427T3 (en)
GR (1) GR3020859T3 (en)
WO (1) WO1993022434A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9279009B2 (en) 2007-12-03 2016-03-08 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services FILIP1L nucleic acid fragments

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US5869248A (en) * 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US6057153A (en) * 1995-01-13 2000-05-02 Yale University Stabilized external guide sequences
US5683873A (en) * 1995-01-13 1997-11-04 Innovir Laboratories, Inc. EGS-mediated inactivation of target RNA
US5877162A (en) * 1996-03-14 1999-03-02 Innovir Laboratories, Inc. Short external guide sequences
US6610478B1 (en) 1996-08-16 2003-08-26 Yale University Phenotypic conversion of cells mediated by external guide sequences
US6013447A (en) * 1997-11-21 2000-01-11 Innovir Laboratories, Inc. Random intracellular method for obtaining optimally active nucleic acid molecules
WO1999027135A2 (en) * 1997-11-21 1999-06-03 Yale University Method for identifying and inhibiting functional nucleic acid molecules in cells
US6248525B1 (en) 1998-03-30 2001-06-19 Yale University Method for identifying essential or functional genes
AU9684601A (en) 2000-10-12 2002-04-22 Univ Rochester Compositions that inhibit proliferation of cancer cells
US7811992B2 (en) 2002-02-06 2010-10-12 Stasys Technologies, Inc. Anti-infarction molecules
EP2116604A1 (en) 2002-08-05 2009-11-11 University of Rochester Protein transducing domain/deaminase chimeric proteins, related compounds, and uses thereof
US8658377B2 (en) 2002-11-15 2014-02-25 Morehouse School Of Medicine Detecting cancer with anti-CCL25 and anti-CCR9 antibodies
US8512701B2 (en) 2002-11-15 2013-08-20 Morehouse School Of Medicine Anti-CXCL13 and anti-CXCR5 antibodies for the prevention and treatment of cancer and cancer cell migration
US9233120B2 (en) 2002-11-15 2016-01-12 Jyant Technologies Anti-CCL25 and anti-CCR9 antibodies for the prevention and treatment of cancer and cancer cell migration
ATE512223T1 (en) 2004-04-29 2011-06-15 Univ Yale NUCLEASE RESISTANT EXTERNAL GUIDANCE SEQUENCES FOR THE TREATMENT OF INFLAMMATORY AND VIRUS RESPIRATORY DISEASES
US7476733B2 (en) 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
EP2395076A1 (en) 2005-10-14 2011-12-14 MUSC Foundation For Research Development Targeting PAX2 for the induction of DEFB1-mediated tumor immunity and cancer therapy
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
AU2007223427A1 (en) 2006-03-01 2007-09-13 University Of Utah Research Foundation Methods and compositions related to cyclic peptide synthesis
US8470965B2 (en) 2006-03-01 2013-06-25 University Of Utah Research Foundation Methods and compositions related to cyclic peptide synthesis
EP3034083B1 (en) 2006-09-21 2020-12-09 University of Rochester Antisense oligonucleotides for use in treating myotonic dystrophy
EP2104516B1 (en) 2006-11-01 2015-01-07 University of Rochester Methods and compositions related to the structure and function of apobec3g
US9896511B2 (en) 2007-01-10 2018-02-20 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Antibodies that bind to TL1A and methods of treating inflammatory or autoimmune disease comprising administering such antibodies
US9822364B2 (en) 2008-03-11 2017-11-21 Yale University Compositions and methods for controlled delivery of inhibitory ribonucleic acids
WO2009114614A2 (en) 2008-03-11 2009-09-17 Yale University Compositions and methods for controlled delivery of inhibitory ribonucleic acids
WO2009137686A1 (en) 2008-05-08 2009-11-12 University Of Utah Research Foundation Sensory receptors for chronic fatigue and pain and uses thereof
US20120070443A1 (en) 2008-12-02 2012-03-22 University Of Utah Research Foundation Pde1 as a target therapeutic in heart disease
WO2010074924A1 (en) 2008-12-23 2010-07-01 University Of Utah Research Foundation Identification and regulation of a novel dna demethylase system
WO2010151638A1 (en) 2009-06-25 2010-12-29 Medical College Of Georgia Research Institute, Inc. Jnk inhibitors for use in treating spinal muscular atrophy
US20110060000A1 (en) 2009-09-10 2011-03-10 Maurizio Grimaldi Acridine analogs in the treatment of gliomas
US20110274745A1 (en) 2009-11-10 2011-11-10 Lipella Pharmaceuticals Inc. Instillation of liposomal formulation of sirna and antisense oligonucleotides
US8889615B2 (en) 2009-12-31 2014-11-18 New York University Methods for promoting epithelialization and healing of chronic wounds
US20110207789A1 (en) 2010-02-19 2011-08-25 Ye Fang Methods related to casein kinase ii (ck2) inhibitors and the use of purinosome-disrupting ck2 inhibitors for anti-cancer therapy agents
CN103608680A (en) 2010-12-14 2014-02-26 詹姆士·W·里拉尔德 Use of anti-CXCL13 antibody and anti-CXCR5 antibody in treatment or detection of malignant tumors
AU2012259312A1 (en) 2011-05-20 2013-12-12 Government Of The United States, As Represented By The Secretary Department Of Health And Human Services Blockade of tl1a-dr3 interactions to ameliorate t cell mediated disease pathology and antibodies thereof
US9801948B2 (en) 2011-09-21 2017-10-31 Yale University Antimicrobial compositions and methods of use thereof
KR101343959B1 (en) 2012-09-19 2013-12-24 한국기계연구원 Integrated coating system
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JP2750127B2 (en) * 1988-07-29 1998-05-13 三共株式会社 Method for Cleavage of Specific Site of Polyribonucleotide by Ribozyme

Patent Citations (1)

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WO1992003566A1 (en) * 1990-08-17 1992-03-05 Yale University Therapeutic ribozyme compositions

Non-Patent Citations (3)

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Title
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA vol. 89, 15 April 1992, WASHINGTON US pages 3185 - 3189 LI, Y. ET AL. 'Targeted cleavage of mRNA in vitro by RNase P from Escherichia coli' cited in the application *
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SCIENCE vol. 249, 17 August 1990, LANCASTER, PA US pages 783 - 786 FORSTER, A. & ALTMAN, S. 'External guide sequences for an RNA enzyme' *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9279009B2 (en) 2007-12-03 2016-03-08 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services FILIP1L nucleic acid fragments

Also Published As

Publication number Publication date
EP0638121A1 (en) 1995-02-15
CA2117903C (en) 1999-01-12
EP0638121B1 (en) 1996-07-17
JPH07507683A (en) 1995-08-31
DE69303712T2 (en) 1997-02-20
CA2117903A1 (en) 1993-11-11
ATE140482T1 (en) 1996-08-15
DK0638121T3 (en) 1996-08-12
JP3015463B2 (en) 2000-03-06
WO1993022434A2 (en) 1993-11-11
DE69303712D1 (en) 1996-08-22
ES2093427T3 (en) 1996-12-16
AU4119893A (en) 1993-11-29
GR3020859T3 (en) 1996-11-30
AU669367B2 (en) 1996-06-06

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