WO2002053581A3 - Spliceosome mediated rna trans-splicing - Google Patents

Spliceosome mediated rna trans-splicing Download PDF

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Publication number
WO2002053581A3
WO2002053581A3 PCT/US2002/000416 US0200416W WO02053581A3 WO 2002053581 A3 WO2002053581 A3 WO 2002053581A3 US 0200416 W US0200416 W US 0200416W WO 02053581 A3 WO02053581 A3 WO 02053581A3
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WO
WIPO (PCT)
Prior art keywords
trans
mrna
splicing
molecules
cells
Prior art date
Application number
PCT/US2002/000416
Other languages
French (fr)
Other versions
WO2002053581A2 (en
Inventor
Lloyd G Mitchell
Mariano A Garcia-Blanco
Carl C Baker
Madaiah Puttaraju
Gary S Mansfield
Hengjun Chao
Original Assignee
Intronn Inc
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 US09/756,097 external-priority patent/US20060088938A1/en
Priority claimed from US09/756,096 external-priority patent/US20030077754A1/en
Priority claimed from US09/756,095 external-priority patent/US20020115207A1/en
Priority claimed from US09/838,858 external-priority patent/US20030148937A1/en
Priority claimed from US09/941,492 external-priority patent/US20030027250A1/en
Priority to EP02714709A priority Critical patent/EP1358203A4/en
Priority to IL15666502A priority patent/IL156665A0/en
Priority to CA002434118A priority patent/CA2434118A1/en
Application filed by Intronn Inc filed Critical Intronn Inc
Priority to AU2002246959A priority patent/AU2002246959B2/en
Priority to IL16505602A priority patent/IL165056A0/en
Priority to JP2002555104A priority patent/JP2004525618A/en
Publication of WO2002053581A2 publication Critical patent/WO2002053581A2/en
Publication of WO2002053581A3 publication Critical patent/WO2002053581A3/en
Priority to IL16505704A priority patent/IL165057A0/en
Priority to AU2004237884A priority patent/AU2004237884B2/en

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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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (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

The molecules and methods of the present invention provide a means for in vivo production of a trans-spliced molecule in a selected subset of cells. The pre-trans-splicing molecules of the invention are substrates for a trans-splicing reaction between the pre-trans-splicing molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides a novel mRNA which is functional as mRNA or encodes a protein to be expressed in the target cells. The expression product of the mRNA is a protein of therapeutic value to the cell or host organism a toxin which causes killing of the specific cells or a novel protein not normally present in such cells. The invention further provides PTMs that have been genetically engineered for the identification of exon/intron boundaries of pre-mRNA molecules using an exon tagging method. The PTMs of the invention can also be designed to result in the production of chimeric RNA encoding for peptide affinity purification tags which can be used to purify and identify proteins expressed in a specific cell type.
PCT/US2002/000416 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing WO2002053581A2 (en)

Priority Applications (8)

Application Number Priority Date Filing Date Title
JP2002555104A JP2004525618A (en) 2001-01-08 2002-01-08 RNA trans-splicing mediated by spliceosomes
IL16505602A IL165056A0 (en) 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing
AU2002246959A AU2002246959B2 (en) 2001-01-08 2002-01-08 Spliceosome mediated RNA trans-splicing
EP02714709A EP1358203A4 (en) 2001-01-08 2002-01-08 Methods and compositions for use in spliceosome mediated rna trans-splicing
CA002434118A CA2434118A1 (en) 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing
IL15666502A IL156665A0 (en) 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing
IL16505704A IL165057A0 (en) 2001-01-08 2004-11-04 Spliceosome mediated rna trans-splicing
AU2004237884A AU2004237884B2 (en) 2001-01-08 2004-12-13 Spliceosome mediated RNA trans-splicing

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US09/756,095 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
US09/756,097 2001-01-08
US09/756,096 2001-01-08
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
US09/838,858 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

Publications (2)

Publication Number Publication Date
WO2002053581A2 WO2002053581A2 (en) 2002-07-11
WO2002053581A3 true WO2002053581A3 (en) 2002-09-26

Family

ID=27542170

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2002/000416 WO2002053581A2 (en) 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing

Country Status (6)

Country Link
EP (1) EP1358203A4 (en)
JP (3) JP2004525618A (en)
AU (1) AU2002246959B2 (en)
CA (1) CA2434118A1 (en)
IL (3) IL165056A0 (en)
WO (1) WO2002053581A2 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10139492B4 (en) 2001-08-13 2004-06-09 Eul, Joachim, Dr. Process for the repair of a mutated RNA from a gene-defective DNA and for the targeted killing of tumor cells by RNA trans-splicing as well as process for the detection of naturally trans-spliced cellular RNA
EP1521766B1 (en) * 2002-06-05 2012-11-07 VIRxSYS Corporation SPLICEOSOME MEDIATED RNA i TRANS /i -SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING
US20060177933A1 (en) * 2004-01-23 2006-08-10 Madaiah Puttaraju Expression of apoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
US7879321B2 (en) * 2004-10-08 2011-02-01 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
WO2006042232A2 (en) * 2004-10-08 2006-04-20 Intronn, Inc. Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
JP5081462B2 (en) * 2007-02-02 2012-11-28 富士フイルム株式会社 Fusion protein production method by trans-splicing method
WO2013025461A1 (en) * 2011-08-12 2013-02-21 Virxsys Corporation Compositions and methods for inducing apoptosis
CN106574270B (en) * 2014-07-03 2021-07-13 豪夫迈·罗氏有限公司 Polypeptide expression system

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6013487A (en) * 1995-12-15 2000-01-11 Mitchell; Lloyd G. Chimeric RNA molecules generated by trans-splicing

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6280978B1 (en) * 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6013487A (en) * 1995-12-15 2000-01-11 Mitchell; Lloyd G. Chimeric RNA molecules generated by trans-splicing

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JONES ET AL.: "Tagging ribozyme reaction sites to follow trans-splicing in mammalian cells", NATURE MEDICINE, vol. 2, no. 6, June 1996 (1996-06-01), pages 643, XP002951116 *
PHYLACTOU ET AL.: "Ribozyme-mediated trans-splicing of a trinucleotide repeat", NATURE GENETICS, vol. 18, no. 4, April 1998 (1998-04-01), pages 378, XP002951115 *

Also Published As

Publication number Publication date
EP1358203A4 (en) 2006-11-22
JP2005176849A (en) 2005-07-07
JP2005168509A (en) 2005-06-30
EP1358203A2 (en) 2003-11-05
IL165056A0 (en) 2005-12-18
IL156665A0 (en) 2004-01-04
CA2434118A1 (en) 2002-07-11
WO2002053581A2 (en) 2002-07-11
AU2002246959B2 (en) 2008-03-06
JP2004525618A (en) 2004-08-26
IL165057A0 (en) 2005-12-18

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