CA2434118A1 - Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage - Google Patents

Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage Download PDF

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Publication number
CA2434118A1
CA2434118A1 CA002434118A CA2434118A CA2434118A1 CA 2434118 A1 CA2434118 A1 CA 2434118A1 CA 002434118 A CA002434118 A CA 002434118A CA 2434118 A CA2434118 A CA 2434118A CA 2434118 A1 CA2434118 A1 CA 2434118A1
Authority
CA
Canada
Prior art keywords
nucleic acid
acid molecule
cell
mrna
target
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
CA002434118A
Other languages
English (en)
Inventor
Lloyd G. Mitchell
Mariano A. Garcia-Blanco
Madaiah Puttaraju
Gary S. Mansfield
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.)
US Department of Health and Human Services
VirxSys Corp
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 US09/756,095 external-priority patent/US20020115207A1/en
Priority claimed from US09/756,096 external-priority patent/US20030077754A1/en
Priority claimed from US09/756,097 external-priority patent/US20060088938A1/en
Priority claimed from US09/838,858 external-priority patent/US20030148937A1/en
Priority claimed from US09/941,492 external-priority patent/US20030027250A1/en
Application filed by Individual filed Critical Individual
Priority to CA002558640A priority Critical patent/CA2558640A1/fr
Publication of CA2434118A1 publication Critical patent/CA2434118A1/fr
Abandoned 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1027—Mutagenizing nucleic acids by DNA shuffling, e.g. RSR, STEP, RPR
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/108—Plasmid DNA episomal vectors
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Microbiology (AREA)
  • Transplantation (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

Cette invention concerne des molécules et des méthodes permettant de produire <i>in vivo</i> une molécule trans-épissée dans un sous-ensemble donné de cellules. Les molécules pré-transépissage selon l'invention sont des substrats pour réaction de trans-épissage entre les molécules pré-transépissage et le pré-ARNm qui est exprimé de manière unique dans les cellules cibles spécifiques. La réaction de trans-épissage <i>in vivo</i> produit un nouvel ARNm fonctionnel en tant que tel ou qui code pour une protéine à exprimer dans les cellules cibles. Le produit d'expression de l'ARNm est une protéine à valeur thérapeutique pour la cellule ou organisme hôte, une toxine qui provoque la mort de cellules spécifiques ou une nouvelle protéine qui n'est normalement pas présente dans de telles cellules. Cette invention concerne également des molécules pré-transépissage (PTM) créées génétiquement pour l'identification de frontières exon/intron de molécules de pré-ARNm au moyen d'une technique de marquage des introns. Les PTM de l'invention peuvent également être conçues en vue de la production d'ARN chimère codant pour des tags de purification à affinité peptidique utilisables pour la purification ou l'identification de protéines exprimées dans un type de cellule spécifique
CA002434118A 2001-01-08 2002-01-08 Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage Abandoned CA2434118A1 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA002558640A CA2558640A1 (fr) 2001-01-08 2002-01-08 Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US09/756,095 US20020115207A1 (en) 1995-12-15 2001-01-08 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US09/756,096 US20030077754A1 (en) 1995-12-15 2001-01-08 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US09/756,095 2001-01-08
US09/756,096 2001-01-08
US09/756,097 2001-01-08
US09/756,097 US20060088938A1 (en) 1995-12-15 2001-01-08 Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
US09/838,858 US20030148937A1 (en) 1995-12-15 2001-04-20 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US09/838,858 2001-04-20
US09/941,492 2001-08-29
US09/941,492 US20030027250A1 (en) 1995-12-15 2001-08-29 Methods and compositions for use in spliceosome mediated RNA trans-splicing
PCT/US2002/000416 WO2002053581A2 (fr) 2001-01-08 2002-01-08 Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA002558640A Division CA2558640A1 (fr) 2001-01-08 2002-01-08 Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage

Publications (1)

Publication Number Publication Date
CA2434118A1 true CA2434118A1 (fr) 2002-07-11

Family

ID=27542170

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002434118A Abandoned CA2434118A1 (fr) 2001-01-08 2002-01-08 Procedes et compositions s'utilisant dans le trans-epissage d'arn induit par complexe d'epissage

Country Status (6)

Country Link
EP (1) EP1358203A4 (fr)
JP (3) JP2004525618A (fr)
AU (1) AU2002246959B2 (fr)
CA (1) CA2434118A1 (fr)
IL (3) IL156665A0 (fr)
WO (1) WO2002053581A2 (fr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10139492B4 (de) 2001-08-13 2004-06-09 Eul, Joachim, Dr. Verfahren zur Reparatur einer mutierten RNA aus einer gendefekten DNA und zum gezielten Abtöten von Tumorzellen durch RNA-Transspleißen sowie Verfahren zum Nachweis von natürlich-transgespleißter zellulärer RNA
WO2003104412A2 (fr) 2002-06-05 2003-12-18 Intronn, Inc. Trans-epissage d'arn a mediation par spliceosome (technique smart) et correction de defauts genetiques de facteur viii a l'aide du trans-epissage d'arn a mediation par spliceosome
EP1716165A4 (fr) * 2004-01-23 2008-06-18 Virxsys Corp Expression d'apoliproteine a1 (apoa-1) et variants au moyen de transepissage d'arn induit par complexe d'epissage
JP5017118B2 (ja) * 2004-10-08 2012-09-05 バークシス コーポレーション 組換えタンパク質をインビボ生産するための、非常に豊富な転写産物の標的化トランススプライシング
AU2005326784B2 (en) * 2004-10-08 2012-03-15 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
JP5081462B2 (ja) * 2007-02-02 2012-11-28 富士フイルム株式会社 トランススプライシング法による融合タンパク質作製方法
JP2014525754A (ja) * 2011-08-12 2014-10-02 バイレクシス コーポレイション アポトーシスを誘導するための組成物および方法
US10308944B2 (en) * 2014-07-03 2019-06-04 Genentech, Inc. Polypeptide expression systems

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0883344B2 (fr) * 1995-12-15 2010-06-09 VIRxSYS Corporation Molecules therapeutiques produites par trans-epissage
US6280978B1 (en) * 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing

Also Published As

Publication number Publication date
WO2002053581A3 (fr) 2002-09-26
EP1358203A4 (fr) 2006-11-22
JP2005168509A (ja) 2005-06-30
JP2004525618A (ja) 2004-08-26
JP2005176849A (ja) 2005-07-07
IL165057A0 (en) 2005-12-18
EP1358203A2 (fr) 2003-11-05
AU2002246959B2 (en) 2008-03-06
IL165056A0 (en) 2005-12-18
IL156665A0 (en) 2004-01-04
WO2002053581A2 (fr) 2002-07-11

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