EP1358203A4 - METHOD AND COMPOSITION FOR CONNECTING IN SPLICEOSOME-MEDIATED RNA TRANS SPLICING - Google Patents

METHOD AND COMPOSITION FOR CONNECTING IN SPLICEOSOME-MEDIATED RNA TRANS SPLICING

Info

Publication number
EP1358203A4
EP1358203A4 EP02714709A EP02714709A EP1358203A4 EP 1358203 A4 EP1358203 A4 EP 1358203A4 EP 02714709 A EP02714709 A EP 02714709A EP 02714709 A EP02714709 A EP 02714709A EP 1358203 A4 EP1358203 A4 EP 1358203A4
Authority
EP
European Patent Office
Prior art keywords
splicing
compositions
methods
mediated rna
rna trans
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.)
Withdrawn
Application number
EP02714709A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP1358203A2 (en
Inventor
Lloyd G Mitchell
Mariano A Garcia-Blanco
Carl C Baker
Madaiah Puttaraju
Gary S Mansfield
Hengjun Chao
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.)
VirxSys Corp
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 US09/756,095 external-priority patent/US20020115207A1/en
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 US09/838,858 external-priority patent/US20030148937A1/en
Priority claimed from US09/941,492 external-priority patent/US20030027250A1/en
Application filed by INTRONN Inc filed Critical INTRONN Inc
Publication of EP1358203A2 publication Critical patent/EP1358203A2/en
Publication of EP1358203A4 publication Critical patent/EP1358203A4/en
Withdrawn legal-status Critical Current

Links

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
    • C12N15/63—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

Landscapes

  • 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)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Molecular Biology (AREA)
  • Biophysics (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)
EP02714709A 2001-01-08 2002-01-08 METHOD AND COMPOSITION FOR CONNECTING IN SPLICEOSOME-MEDIATED RNA TRANS SPLICING Withdrawn EP1358203A4 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US941492 1986-12-15
US756096 1991-09-06
US756097 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
US756095 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
US09/756,096 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
US838858 2001-04-20
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 (en) 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing

Publications (2)

Publication Number Publication Date
EP1358203A2 EP1358203A2 (en) 2003-11-05
EP1358203A4 true EP1358203A4 (en) 2006-11-22

Family

ID=27542170

Family Applications (1)

Application Number Title Priority Date Filing Date
EP02714709A Withdrawn EP1358203A4 (en) 2001-01-08 2002-01-08 METHOD AND COMPOSITION FOR CONNECTING IN SPLICEOSOME-MEDIATED RNA TRANS SPLICING

Country Status (6)

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

Families Citing this family (10)

* 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 (en) 2002-06-05 2003-12-18 Intronn, Inc. Spliceosome mediated rna trans-splicing and correction of factor viii genetic defects using spliceosome mediated rna trans spling
AU2005207053A1 (en) * 2004-01-23 2005-08-04 Virxsys Corporation Expression of ApoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
WO2006042232A2 (en) * 2004-10-08 2006-04-20 Intronn, Inc. Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
CA2583306A1 (en) * 2004-10-08 2006-08-10 Intronn, Inc. 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
EP2742128A4 (en) * 2011-08-12 2015-03-18 Virxsys Corp COMPOSITIONS AND METHODS FOR APOPTOSIS INDUCTION
US10308944B2 (en) * 2014-07-03 2019-06-04 Genentech, Inc. Polypeptide expression systems
WO2022067228A1 (en) * 2020-09-28 2022-03-31 U1 Bio, Inc. Trans-splicing system for tissue-specific replacement of rna sequences
WO2022220968A1 (en) * 2021-04-15 2022-10-20 U1 Bio, Inc. High efficiency trans-splicing for replacement of targeted rna sequences in human cells

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000009734A2 (en) * 1998-08-13 2000-02-24 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated rna trans-splicing

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU730305B2 (en) * 1995-12-15 2001-03-01 Intronn Llc Therapeutic molecules generated by trans-splicing

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000009734A2 (en) * 1998-08-13 2000-02-24 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated rna trans-splicing

Also Published As

Publication number Publication date
AU2002246959B2 (en) 2008-03-06
JP2005168509A (ja) 2005-06-30
IL156665A0 (en) 2004-01-04
EP1358203A2 (en) 2003-11-05
JP2005176849A (ja) 2005-07-07
CA2434118A1 (en) 2002-07-11
JP2004525618A (ja) 2004-08-26
WO2002053581A3 (en) 2002-09-26
IL165056A0 (en) 2005-12-18
WO2002053581A2 (en) 2002-07-11
IL165057A0 (en) 2005-12-18

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Legal Events

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PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

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Effective date: 20030808

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AX Request for extension of the european patent

Extension state: AL LT LV MK RO SI

RIN1 Information on inventor provided before grant (corrected)

Inventor name: CHAO, HENGJUN

Inventor name: MANSFIELD, GARY, S.

Inventor name: PUTTARAJU, MADAIAH

Inventor name: BAKER, CARL, C.

Inventor name: GARCIA-BLANCO, MARIANO, A.

Inventor name: MITCHELL, LLOYD, G.

A4 Supplementary search report drawn up and despatched

Effective date: 20061020

RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 15/85 20060101AFI20061017BHEP

17Q First examination report despatched

Effective date: 20070212

RAP1 Party data changed (applicant data changed or rights of an application transferred)

Owner name: VIRXSYS CORPORATION

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Effective date: 20090825