KR20120094166A - 유전자 치료 적용을 위한 초나선화된 미니서클 ｄｎａ - Google Patents

유전자 치료 적용을 위한 초나선화된 미니서클 ｄｎａ Download PDF

Info

Publication number
KR20120094166A
KR20120094166A KR20127012633A KR20127012633A KR20120094166A KR 20120094166 A KR20120094166 A KR 20120094166A KR 20127012633 A KR20127012633 A KR 20127012633A KR 20127012633 A KR20127012633 A KR 20127012633A KR 20120094166 A KR20120094166 A KR 20120094166A
Authority
KR
South Korea
Prior art keywords
nucleic acid
minivector
acid sequence
gene
dna
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Withdrawn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
KR20127012633A
Other languages
English (en)
Korean (ko)
Inventor
이. 린 제키드리치
조나단 포그
주니어 다니엘 제임스 카타니즈
에롤 바칼바시
브라이언 이. 길버트
Original Assignee
베일러 칼리지 오브 메디신
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
Application filed by 베일러 칼리지 오브 메디신 filed Critical 베일러 칼리지 오브 메디신
Publication of KR20120094166A publication Critical patent/KR20120094166A/ko
Withdrawn legal-status Critical Current

Links

Images

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
    • 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
    • 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
    • C12N15/111—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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
KR20127012633A 2009-10-16 2010-10-15 유전자 치료 적용을 위한 초나선화된 미니서클 ｄｎａ Withdrawn KR20120094166A (ko)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US25245509P 2009-10-16 2009-10-16
US61/252,455 2009-10-16

Publications (1)

Publication Number Publication Date
KR20120094166A true KR20120094166A (ko) 2012-08-23

Family

ID=43876905

Family Applications (1)

Application Number Title Priority Date Filing Date
KR20127012633A Withdrawn KR20120094166A (ko) 2009-10-16 2010-10-15 유전자 치료 적용을 위한 초나선화된 미니서클 ｄｎａ

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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP2801614B1 (en) * 2002-08-29 2019-05-15 The Board of Trustees of the Leland Stanford Junior University Circular nucleic acid vectors and methods for making and using the same
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
AU2006204120A1 (en) * 2005-01-06 2006-07-13 Benitec, Inc. RNAi agents for maintenance of stem cells
US7622252B2 (en) * 2005-06-10 2009-11-24 Baylor College Of Medicine Generation of minicircle DNA with physiological supercoiling
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

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
AU2010306645B2 (en) 2017-03-23
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

Similar Documents

Publication Publication Date Title
AU2021201683B2 (en) Novel CAS13B orthologues CRISPR enzymes and systems
US8460924B2 (en) Supercoiled minivectors™ for gene therapy applications
US20230365951A1 (en) Tracking and Manipulating Cellular RNA via Nuclear Delivery of CRISPR/CAS9
KR102812752B1 (ko) Rna-가이드된 핵산 변형 효소 및 이의 사용 방법
JP5066095B2 (ja) 染色体ｄｎａに標的化されるオリゴマーによる遺伝子発現の調節
WO2019089820A1 (en) Casz compositions and methods of use
US20130245096A1 (en) COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA
KR20200006054A (ko) 신규 타입 vi crispr 오르소로그 및 시스템
US11884918B2 (en) Systems and methods for modulating CRISPR activity
US20250049960A1 (en) Multicomponent systems for site-specific genome modifications
Tou et al. Immune evasive DNA donors and recombinases license kilobase-scale writing
US20130085173A1 (en) Supercoiled minicircle dna for gene therapy applications
JP6795492B2 (ja) ＣＬＣＮ７（ＡＤＯ２ ＣＬＣＮ７依存性）遺伝子変異によって引き起こされる常染色体優性大理石骨病２型（ＡＤＯ２）療法のための短鎖干渉ＲＮＡ（ｓｉＲＮＡ）
HK40024463A (en) Novel type vi crispr orthologs and systems
Curtis et al. Developing gene knockdown-replacement therapies for spinocerebellar ataxia type 7

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20120516

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1203 Withdrawal of no request for examination
WITN Application deemed withdrawn, e.g. because no request for examination was filed or no examination fee was paid