AU2010306645B2 - Supercoiled minicircle DNA for gene therapy applications - Google Patents

Supercoiled minicircle DNA for gene therapy applications Download PDF

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Publication number
AU2010306645B2
AU2010306645B2 AU2010306645A AU2010306645A AU2010306645B2 AU 2010306645 B2 AU2010306645 B2 AU 2010306645B2 AU 2010306645 A AU2010306645 A AU 2010306645A AU 2010306645 A AU2010306645 A AU 2010306645A AU 2010306645 B2 AU2010306645 B2 AU 2010306645B2
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Australia
Prior art keywords
minivector
dna
cell
gene
nucleic acid
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Ceased
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AU2010306645A
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AU2010306645A1 (en
Inventor
Erol Bakkalbasi
Daniel James Catanese Jr.
Jonathan Fogg
Brian E. Gilbert
E. Lynn Zechiedrich
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Baylor College of Medicine
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Baylor College of Medicine
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    • 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
    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2010306645A 2009-10-16 2010-10-15 Supercoiled minicircle DNA for gene therapy applications Ceased AU2010306645B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US25245509P 2009-10-16 2009-10-16
US61/252,455 2009-10-16
PCT/US2010/052920 WO2011047318A2 (en) 2009-10-16 2010-10-15 Supercoiled minicircle dna for gene therapy applications

Publications (2)

Publication Number Publication Date
AU2010306645A1 AU2010306645A1 (en) 2012-06-07
AU2010306645B2 true AU2010306645B2 (en) 2017-03-23

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AU2010306645A Ceased AU2010306645B2 (en) 2009-10-16 2010-10-15 Supercoiled minicircle DNA for gene therapy applications

Country Status (10)

Country Link
US (3) US8460924B2 (enExample)
EP (1) EP2488641B1 (enExample)
JP (2) JP2013507934A (enExample)
KR (1) KR20120094166A (enExample)
AU (1) AU2010306645B2 (enExample)
BR (1) BR112012011576A2 (enExample)
CA (1) CA2777835A1 (enExample)
MX (1) MX2012004447A (enExample)
NZ (1) NZ599998A (enExample)
WO (1) WO2011047318A2 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101510830B1 (ko) 2012-05-11 2015-04-10 가톨릭대학교 산학협력단 생리활성 단백질을 발현하는 미니서클을 이용하여 형질전환된 줄기세포를 통한 치료법
WO2013181440A1 (en) * 2012-05-30 2013-12-05 Baylor College Of Medicine Supercoiled minivectors as a tool for dna repair, alteration and replacement
EP2735614A1 (en) * 2012-11-22 2014-05-28 Centre National De La Recherche Scientifique In vitro production of DNA minicircles comprising less than 250 base pairs
US10604771B2 (en) 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
GB201702500D0 (en) * 2017-02-16 2017-04-05 Univ Sheffield Stable vamp reporter assay
US20180305701A1 (en) 2017-03-14 2018-10-25 Baylor College Of Medicine Targeting minivectors to specific tissue using shape
US20200048716A1 (en) 2017-11-03 2020-02-13 Twister Biotech, Inc Using minivectors to treat ovarian cancer
WO2021055760A1 (en) 2019-09-18 2021-03-25 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
US20240100189A1 (en) 2021-09-10 2024-03-28 Twister Biotech, Inc Using minivectors to treat idiopathic pulmonary fibrosis
US20240093226A1 (en) 2021-09-10 2024-03-21 Baylor College Of Medicine Ultrapure minivectors for gene therapy
US20230190955A1 (en) 2021-10-21 2023-06-22 Baylor College Of Medicine Treatment of liver cancer or liver fibrosis

Citations (1)

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WO2004020605A2 (en) * 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same

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FR2626584B1 (fr) * 1988-01-28 1990-07-13 Agronomique Inst Nat Rech Sequence ars efficace chez yarrowia lipolytica et procede pour sa preparation
WO1994009127A2 (en) 1992-10-16 1994-04-28 The Government Of The United States Of America As Represented By The Secretary, Department Of Healthand Human Services Supercoiled minicircle dna as as a unitary promoter vector
US6964861B1 (en) * 1998-11-13 2005-11-15 Invitrogen Corporation Enhanced in vitro recombinational cloning of using ribosomal proteins
EP2105496B1 (en) 2000-12-08 2013-02-20 Life Technologies Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
GB0108968D0 (en) * 2001-04-10 2001-05-30 Imp College Innovations Ltd Methods
WO2002083889A2 (en) 2001-04-10 2002-10-24 Imperial College Innovations Limited Methods for the production of minicircles
WO2003064623A2 (en) 2002-01-31 2003-08-07 Washington State University Research Foundation Methods and vectors for facilitating site-specific recombination
AU2003273995A1 (en) 2002-06-05 2003-12-22 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
US7288529B2 (en) * 2003-04-24 2007-10-30 New York University ALK protein tyrosine kinase, cells and methods embodying and using same
ATE340264T1 (de) * 2003-06-10 2006-10-15 Mologen Ag Zirkuläres expressionskonstrukt für gentherapeutische anwendungen
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WO2007130604A2 (en) * 2006-05-04 2007-11-15 Baylor Research Institute Anti-tumor activity of an oncolytic adenovirus-delivered oncogene sirna
EP2178896A1 (en) 2007-07-31 2010-04-28 The Johns Hopkins University Polypeptide-nucleic acid conjugate for immunoprophylaxis or immunotherapy for neoplastic or infectious disorders

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2004020605A2 (en) * 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same

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Also Published As

Publication number Publication date
US20110160284A1 (en) 2011-06-30
WO2011047318A2 (en) 2011-04-21
EP2488641A2 (en) 2012-08-22
US8729044B2 (en) 2014-05-20
US20130316449A1 (en) 2013-11-28
WO2011047318A3 (en) 2011-09-09
US20120302625A1 (en) 2012-11-29
MX2012004447A (es) 2012-10-15
JP2013507934A (ja) 2013-03-07
US9267150B2 (en) 2016-02-23
EP2488641A4 (en) 2013-11-13
BR112012011576A2 (pt) 2015-09-15
EP2488641B1 (en) 2019-01-23
US8460924B2 (en) 2013-06-11
KR20120094166A (ko) 2012-08-23
NZ599998A (en) 2014-11-28
CA2777835A1 (en) 2011-04-21
AU2010306645A1 (en) 2012-06-07
WO2011047318A8 (en) 2011-06-09
JP2017046710A (ja) 2017-03-09

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