AU2003215249B2 - Methods and compositions for use in spliceosome mediated RNA trans-splicing - Google Patents

Methods and compositions for use in spliceosome mediated RNA trans-splicing Download PDF

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Publication number
AU2003215249B2
AU2003215249B2 AU2003215249A AU2003215249A AU2003215249B2 AU 2003215249 B2 AU2003215249 B2 AU 2003215249B2 AU 2003215249 A AU2003215249 A AU 2003215249A AU 2003215249 A AU2003215249 A AU 2003215249A AU 2003215249 B2 AU2003215249 B2 AU 2003215249B2
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AU
Australia
Prior art keywords
nucleic acid
mrna
acid molecule
target
cell
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
AU2003215249A
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English (en)
Other versions
AU2003215249A1 (en
Inventor
Mariano A. Garcia-Blanco
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
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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 US10/075,028 external-priority patent/US20030153054A1/en
Priority claimed from US10/076,248 external-priority patent/US20020193580A1/en
Application filed by VirxSys Corp filed Critical VirxSys Corp
Publication of AU2003215249A1 publication Critical patent/AU2003215249A1/en
Assigned to VIRXSYS CORPORATION reassignment VIRXSYS CORPORATION Request for Assignment Assignors: INTRONN, INC.
Application granted granted Critical
Publication of AU2003215249B2 publication Critical patent/AU2003215249B2/en
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
    • C12N15/11—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pulmonology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Biochemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
AU2003215249A 2002-02-12 2003-02-12 Methods and compositions for use in spliceosome mediated RNA trans-splicing Ceased AU2003215249B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US10/075,028 2002-02-12
US10/075,028 US20030153054A1 (en) 2002-02-12 2002-02-12 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US10/076,248 2002-02-12
US10/076,248 US20020193580A1 (en) 1995-12-15 2002-02-12 Methods and compositions for use in spliceosome mediated RNA trans-splicing
PCT/US2003/004622 WO2003069311A2 (en) 2002-02-12 2003-02-12 Methods and compositions for use in spliceosome mediated rna trans-splicing

Publications (2)

Publication Number Publication Date
AU2003215249A1 AU2003215249A1 (en) 2003-09-04
AU2003215249B2 true AU2003215249B2 (en) 2008-07-03

Family

ID=27736827

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2003215249A Ceased AU2003215249B2 (en) 2002-02-12 2003-02-12 Methods and compositions for use in spliceosome mediated RNA trans-splicing

Country Status (5)

Country Link
EP (1) EP1539788A2 (de)
JP (1) JP2006505242A (de)
AU (1) AU2003215249B2 (de)
CA (1) CA2475802A1 (de)
WO (1) WO2003069311A2 (de)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007518423A (ja) * 2004-01-23 2007-07-12 イントロン、インコーポレイテッド スプライセオソーム仲介型ｒｎａトランススプライシングを使用するアポａ−１及びその変異体の発現
EP2151248A1 (de) 2008-07-30 2010-02-10 Johann Bauer Verbesserte Prä-mRNA-Trans-Splicing-Moleküle und deren Verwendungen
EP3377116A4 (de) 2015-11-19 2019-07-10 The Trustees of The University of Pennsylvania Zusammensetzungen und verfahren zur korrektur von erblicher augenerkrankung
CA3097004A1 (en) 2018-04-17 2019-10-24 The Trustees Of The University Of Pennsylvania Trans-splicing molecules
AU2023269865A1 (en) 2022-05-13 2024-11-21 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules

Citations (2)

* 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
US6083702A (en) * 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing

Patent Citations (2)

* 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
US6083702A (en) * 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing

Also Published As

Publication number Publication date
JP2006505242A (ja) 2006-02-16
CA2475802A1 (en) 2003-08-21
WO2003069311A3 (en) 2005-04-21
WO2003069311A2 (en) 2003-08-21
AU2003215249A1 (en) 2003-09-04
EP1539788A2 (de) 2005-06-15

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Legal Events

Date Code Title Description
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