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WO1998039427A3 - Gene therapy using bone marrow transplants transfected with therapeutic genes under the control of tissue-specific promoters - Google Patents

Gene therapy using bone marrow transplants transfected with therapeutic genes under the control of tissue-specific promoters Download PDF

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Publication number
WO1998039427A3
WO1998039427A3 PCT/US1998/004421 US9804421W WO9839427A3 WO 1998039427 A3 WO1998039427 A3 WO 1998039427A3 US 9804421 W US9804421 W US 9804421W WO 9839427 A3 WO9839427 A3 WO 9839427A3
Authority
WO
WIPO (PCT)
Prior art keywords
transfected
tissue
bone marrow
control
gene therapy
Prior art date
Application number
PCT/US1998/004421
Other languages
French (fr)
Other versions
WO1998039427A2 (en
Inventor
Gary S Stein
Janet L Stein
Susie Nilsson
Stefan Peters
Baruch Frenkel
Zhen Hou
Peter Quesenberry
Jane B Lian
Original Assignee
Univ Massachusetts
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 Univ Massachusetts filed Critical Univ Massachusetts
Priority to AU63468/98A priority Critical patent/AU6346898A/en
Publication of WO1998039427A2 publication Critical patent/WO1998039427A2/en
Publication of WO1998039427A3 publication Critical patent/WO1998039427A3/en

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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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0663—Bone marrow mesenchymal stem cells (BM-MSC)
    • 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
    • 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
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Rheumatology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Methods for expressing exogenous genes in differentiated cells of a specific type, such as skeletal tissue cells are disclosed. According to the method, pluripotent stem cells capable of maturing into differentiated cells of a specific type are contacted with a nucleic acid comprising an exogenous gene operatively linked to a regulatory element capable of controlling expression of the gene in the differentiated cell. As a result of the method, a population of transduced stem cells capable of maturing into differentiated cells expressing the exogenous gene is produced.
PCT/US1998/004421 1997-03-06 1998-03-06 Gene therapy using bone marrow transplants transfected with therapeutic genes under the control of tissue-specific promoters WO1998039427A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU63468/98A AU6346898A (en) 1997-03-06 1998-03-06 Gene therapy using bone marrow transplants transfected with therapeutic genes under the control of tissue-specific promoters

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US3983997P 1997-03-06 1997-03-06
US60/039,839 1997-03-06

Publications (2)

Publication Number Publication Date
WO1998039427A2 WO1998039427A2 (en) 1998-09-11
WO1998039427A3 true WO1998039427A3 (en) 1998-12-03

Family

ID=21907604

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/004421 WO1998039427A2 (en) 1997-03-06 1998-03-06 Gene therapy using bone marrow transplants transfected with therapeutic genes under the control of tissue-specific promoters

Country Status (2)

Country Link
AU (1) AU6346898A (en)
WO (1) WO1998039427A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1084278B1 (en) * 1998-06-08 2005-10-19 ACGT Medico Inc. Detection of dna, rna and proteins
DE19850986A1 (en) * 1998-11-05 2000-05-25 Aventis Pharma Gmbh The genetic engineering of cells and their use for the prophylaxis and therapy of diseases
GB2343679A (en) * 1998-11-16 2000-05-17 Alison Miriam Davies Autologous transplantation and method for making cells dormant
AU4771600A (en) * 2000-05-16 2001-11-26 Alison Davies Cells, culture methods and their uses
US6576464B2 (en) 2000-11-27 2003-06-10 Geron Corporation Methods for providing differentiated stem cells
US6921665B2 (en) 2000-11-27 2005-07-26 Roslin Institute (Edinburgh) Selective antibody targeting of undifferentiated stem cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994016715A1 (en) * 1993-01-27 1994-08-04 Hemosol, Inc. Selective cell proliferation
EP0683226A1 (en) * 1994-04-27 1995-11-22 Hoechst Japan Kabushiki Kaisha Transgenic animals as a model for metabolic bone diseases
WO1996005299A1 (en) * 1994-08-15 1996-02-22 Garvan Institute Of Medical Research Dna vector for bone-specific gene expression

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994016715A1 (en) * 1993-01-27 1994-08-04 Hemosol, Inc. Selective cell proliferation
EP0683226A1 (en) * 1994-04-27 1995-11-22 Hoechst Japan Kabushiki Kaisha Transgenic animals as a model for metabolic bone diseases
WO1996005299A1 (en) * 1994-08-15 1996-02-22 Garvan Institute Of Medical Research Dna vector for bone-specific gene expression

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
BORTELL R ET AL: "VITAMIN D-RESPONSIVE PROTEIN-DNA INTERACTIONS AT MULTIPLE PROMOTER REGULATORY ELEMENTS THAT CONTRIBUTE TO THE LEVEL OF RAT OSTEOCALCIN GENE EXPRESSION", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, vol. 89, no. 13, 1 July 1992 (1992-07-01), pages 6119 - 6123, XP000570307 *
CELESTE A J ET AL: "ISOLATION OF THE HUMAN GENE FOR BONE GLA PROTEIN UTILIZING MOUSE AND RAT CDNA CLONES", EMBO JOURNAL., vol. 5, 1986, EYNSHAM, OXFORD GB, pages 1885 - 1890, XP002071377 *
FRENKEL B ET AL.: "ACTIVITY OF THE OSTEOCALCIN PROMOTER IN SKELETAL SITES OF TRANSGENIC MICE AND DURING OSTEOBLAST DIFFERENTIATION IN BONE-MARROW DERIVED STROMAL CELL CULTURES: EFFECTS OF AGE AND SEX", ENDOCRINOLOGY, vol. 138, no. 5, May 1997 (1997-05-01), SPRINGFIELD US, pages 2109 - 2116, XP002071378 *
GRONTHOS S ET AL: "THE STRO-1+ FRACTION OF ADULT HUMAN BONE MARROW CONTAINS THE OSTEOGENIC PRECURSORS", BLOOD, vol. 84, no. 12, 15 December 1994 (1994-12-15), WASHINGTON US, pages 4164 - 4173, XP000616142 *
HOFFMANN H M ET AL.: "TRANSCRIPTIONAL CONTROL OF THE TISSUE-SPECIFIC, DEVELOPMENTALLY REGULATED OSTEOCALCIN GENE REQUIRES A BINDING MOTIF FOR THE MSX FAMILY OF HOMEODOMAIN PROTEINS", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA., vol. 91, 1994, WASHINGTON US, pages 12887 - 12891, XP002071375 *
KESTERSON R A ET AL.: "The human osteocalcin promoter directs bone-specific vatamin D-regulatable gene expression in transgenic mice", MOLECULAR ENDOCRINOLOGY (UNITED STATES), vol. 7, no. 3, 1993, BETHESDA US, pages 462 - 467, XP002071374 *
LIAN J B ET AL: "THE OSTEOCALCIN GENE PROMOTER PROVIDES A MOLECULAR BLUEPRINT FOR REGULATORY MECHANISMS CONTROLLING BONE TISSUE FORMATION: ROLE OF TRANSCRIPTION FACTORS INVOLVED IN DEVELOPMENT", CONNECTIVE TISSUE RESEARCH, vol. 35, no. 1-4, 1996, AMSTERDAM NL, pages 15 - 21, XP002071376 *

Also Published As

Publication number Publication date
WO1998039427A2 (en) 1998-09-11
AU6346898A (en) 1998-09-22

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