AU2001280576A1 - Tissue-specific self-inactivating gene therapy vector - Google Patents

Tissue-specific self-inactivating gene therapy vector

Info

Publication number
AU2001280576A1
AU2001280576A1 AU2001280576A AU8057601A AU2001280576A1 AU 2001280576 A1 AU2001280576 A1 AU 2001280576A1 AU 2001280576 A AU2001280576 A AU 2001280576A AU 8057601 A AU8057601 A AU 8057601A AU 2001280576 A1 AU2001280576 A1 AU 2001280576A1
Authority
AU
Australia
Prior art keywords
tissue
gene therapy
therapy vector
inactivating gene
specific self
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.)
Abandoned
Application number
AU2001280576A
Inventor
David T Curiel
Paul N. Reynolds
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.)
UAB Research Foundation
Original Assignee
UAB Research Foundation
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 UAB Research Foundation filed Critical UAB Research Foundation
Publication of AU2001280576A1 publication Critical patent/AU2001280576A1/en
Abandoned 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
    • C12N15/86—Viral vectors
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
AU2001280576A 2000-07-18 2001-07-17 Tissue-specific self-inactivating gene therapy vector Abandoned AU2001280576A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US21924200P 2000-07-18 2000-07-18
US60219242 2000-07-18
PCT/US2001/022407 WO2002006451A1 (en) 2000-07-18 2001-07-17 Tissue-specific self-inactivating gene therapy vector

Publications (1)

Publication Number Publication Date
AU2001280576A1 true AU2001280576A1 (en) 2002-01-30

Family

ID=22818472

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001280576A Abandoned AU2001280576A1 (en) 2000-07-18 2001-07-17 Tissue-specific self-inactivating gene therapy vector

Country Status (3)

Country Link
US (1) US6716622B2 (en)
AU (1) AU2001280576A1 (en)
WO (1) WO2002006451A1 (en)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0415963D0 (en) * 2004-07-16 2004-08-18 Cxr Biosciences Ltd Detection of cellular stress
CN101203611B (en) * 2005-04-19 2013-08-14 巴斯福植物科学有限公司 Improved methods controlling gene expression
US20080300202A1 (en) * 2006-05-18 2008-12-04 The State of Oregon acting by and through the State Board of Higher Education on behalf of the Subtractive transgenics
US9388425B2 (en) * 2006-10-20 2016-07-12 Trustees Of Boston University Tunable genetic switch for regulating gene expression
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US9375440B2 (en) * 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
CN102002483B (en) * 2010-07-19 2013-04-17 中国人民解放军军事医学科学院放射与辐射医学研究所 Hematopoietic targeted helper adenovirus and application thereof
JP6320382B2 (en) 2012-08-13 2018-05-09 ザ ロックフェラー ユニヴァーシティ Melanoma treatment and diagnosis
ES2782825T3 (en) 2012-10-31 2020-09-16 Univ Rockefeller Metastatic Colon Cancer Treatment
EP2914731B1 (en) * 2012-11-01 2019-09-11 California Institute of Technology Reversible gene expression
CN103981147B (en) 2013-02-08 2017-11-10 中国科学院上海生命科学研究院 A kind of new method for preparing hepatic parenchymal cells
WO2019104062A1 (en) 2017-11-21 2019-05-31 Rgenix, Inc. Polymorphs and uses thereof
US11174220B2 (en) 2019-12-13 2021-11-16 Inspirna, Inc. Metal salts and uses thereof
CA3172591A1 (en) 2020-03-24 2021-09-30 Douglas Anthony KERR Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
US20230138409A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024091824A1 (en) 2022-10-26 2024-05-02 Ada Forsyth Institute, Inc. Differentiation and reprogramming of chondrocyte

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5919676A (en) * 1993-06-24 1999-07-06 Advec, Inc. Adenoviral vector system comprising Cre-loxP recombination
JP4216350B2 (en) * 1994-09-19 2009-01-28 大日本住友製薬株式会社 Recombinant DNA viral vector for animal cell infection
US5712114A (en) * 1995-06-06 1998-01-27 Basf Aktiengesellschaft Compositions for expression of proteins in host cells using a preprocollagen signal sequence

Also Published As

Publication number Publication date
WO2002006451A1 (en) 2002-01-24
US6716622B2 (en) 2004-04-06
US20020022018A1 (en) 2002-02-21

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