AU2002246959B2 - Spliceosome mediated RNA trans-splicing - Google Patents

Spliceosome mediated RNA trans-splicing Download PDF

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Publication number
AU2002246959B2
AU2002246959B2 AU2002246959A AU2002246959A AU2002246959B2 AU 2002246959 B2 AU2002246959 B2 AU 2002246959B2 AU 2002246959 A AU2002246959 A AU 2002246959A AU 2002246959 A AU2002246959 A AU 2002246959A AU 2002246959 B2 AU2002246959 B2 AU 2002246959B2
Authority
AU
Australia
Prior art keywords
target
ptm
splicing
trans
splice
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
AU2002246959A
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English (en)
Other versions
AU2002246959A1 (en
Inventor
Mariano A. Garcia-Blanco
Gary S. Mansfield
Lloyd G. Mitchell
Madaiah Puttaraju
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
VirxSys Corp
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 US09/756,096 external-priority patent/US20030077754A1/en
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 US09/838,858 external-priority patent/US20030148937A1/en
Priority claimed from US09/941,492 external-priority patent/US20030027250A1/en
Application filed by VirxSys Corp filed Critical VirxSys Corp
Publication of AU2002246959A1 publication Critical patent/AU2002246959A1/en
Assigned to THE UNIVERSITY OF NORTH CAROLINA, AT CHAPEL HILL, INTRONN, INC, THE GOVERNMENT, AS REPRESENTED BY THE SECRETARY OF DEPARTMENT OF HEALTH AND HUMAN SERVICES reassignment THE UNIVERSITY OF NORTH CAROLINA, AT CHAPEL HILL Amend patent request/document other than specification (104) Assignors: Refer to Publication History
Priority to AU2004237884A priority Critical patent/AU2004237884B2/en
Assigned to INTRONN, INC reassignment INTRONN, INC Amend patent request/document other than specification (104) Assignors: INTRONN, INC, The University of North Carolina at Chapel Hill and The Government, as represented by the Secretary of Department of Health and Human Services.
Assigned to VIRXSYS CORPORATION reassignment VIRXSYS CORPORATION Request for Assignment Assignors: INTRONN, INC.
Publication of AU2002246959B2 publication Critical patent/AU2002246959B2/en
Application granted granted Critical
Anticipated expiration legal-status Critical
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
    • C12N15/63—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)
  • 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)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Molecular Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Transplantation (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)
AU2002246959A 2001-01-08 2002-01-08 Spliceosome mediated RNA trans-splicing Ceased AU2002246959B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2004237884A AU2004237884B2 (en) 2001-01-08 2004-12-13 Spliceosome mediated RNA trans-splicing

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US09/756,096 US20030077754A1 (en) 1995-12-15 2001-01-08 Methods and compositions for use in spliceosome mediated RNA trans-splicing
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
US09/756,095 US20020115207A1 (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
US09/838,858 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 US20030027250A1 (en) 1995-12-15 2001-08-29 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US09/941,492 2001-08-29
PCT/US2002/000416 WO2002053581A2 (en) 2001-01-08 2002-01-08 Spliceosome mediated rna trans-splicing

Related Child Applications (2)

Application Number Title Priority Date Filing Date
AU2004237883A Division AU2004237883A1 (en) 2001-01-08 2004-12-13 Spliceosome mediated RNA trans-splicing
AU2004237884A Division AU2004237884B2 (en) 2001-01-08 2004-12-13 Spliceosome mediated RNA trans-splicing

Publications (2)

Publication Number Publication Date
AU2002246959A1 AU2002246959A1 (en) 2003-01-23
AU2002246959B2 true AU2002246959B2 (en) 2008-03-06

Family

ID=27542170

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2002246959A Ceased AU2002246959B2 (en) 2001-01-08 2002-01-08 Spliceosome mediated RNA trans-splicing

Country Status (6)

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

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
CA2553828A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. 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
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
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
HK1199285A1 (en) * 2011-08-12 2015-06-26 Virxsys Corporation Compositions and methods for inducing apoptosis
PL3164492T3 (pl) * 2014-07-03 2020-04-30 F. Hoffmann-La Roche Ag Układy ekspresji polipeptydu
EP4217010A4 (en) * 2020-09-28 2025-01-08 Tacit Therapeutics, 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
EP0883344B2 (en) * 1995-12-15 2010-06-09 VIRxSYS Corporation 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
WO2002053581A2 (en) 2002-07-11
WO2002053581A3 (en) 2002-09-26
JP2005168509A (ja) 2005-06-30
JP2004525618A (ja) 2004-08-26
EP1358203A4 (en) 2006-11-22
JP2005176849A (ja) 2005-07-07
IL165057A0 (en) 2005-12-18
IL156665A0 (en) 2004-01-04
EP1358203A2 (en) 2003-11-05
CA2434118A1 (en) 2002-07-11
IL165056A0 (en) 2005-12-18

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DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: ADD CO-APPLICANTS THE UNIVERSITY OF NORTH CAROLINA, AT CHAPEL HILL AND THE GOVERNMENT, AS REPRESENTED BY THE SECRETARY OF DEPARTMENT OF HEALTH AND HUMAN SERVICES.

DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: DELETE CO-INVENTORS CARL C. BAKER AND HENGJUN CHAO.

Free format text: THE NATURE OF THE AMENDMENT IS: DELETE CO-APPLICANTS THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL AND THE GOVERNMENT, AS REPRESENTED BY THE SECRETARY OF DEPARTMENT OF HEALTH AND HUMAN SERVICES.

PC1 Assignment before grant (sect. 113)

Owner name: VIRXSYS CORPORATION

Free format text: FORMER APPLICANT(S): INTRONN, INC.

FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired