WO1999025814B1 - Genetically-modified myogenic progenitors and their use in cell and gene therapy - Google Patents

Genetically-modified myogenic progenitors and their use in cell and gene therapy

Info

Publication number
WO1999025814B1
WO1999025814B1 PCT/EP1998/007263 EP9807263W WO9925814B1 WO 1999025814 B1 WO1999025814 B1 WO 1999025814B1 EP 9807263 W EP9807263 W EP 9807263W WO 9925814 B1 WO9925814 B1 WO 9925814B1
Authority
WO
WIPO (PCT)
Prior art keywords
bone
cells according
marrow derived
precursor cells
coding
Prior art date
Application number
PCT/EP1998/007263
Other languages
French (fr)
Other versions
WO1999025814A1 (en
Inventor
Fulvio Mavilio
Original Assignee
Genera Spa
Fulvio Mavilio
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 Genera Spa, Fulvio Mavilio filed Critical Genera Spa
Priority to CA002310016A priority Critical patent/CA2310016A1/en
Priority to AU18725/99A priority patent/AU1872599A/en
Priority to JP2000521180A priority patent/JP2001523448A/en
Priority to EP98963450A priority patent/EP1030908A1/en
Publication of WO1999025814A1 publication Critical patent/WO1999025814A1/en
Publication of WO1999025814B1 publication Critical patent/WO1999025814B1/en

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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood 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

Abstract

The present invention has as an object engineered myogenic progenitors derived from bone marrow, useful in muscular dystrophy gene therapy.

Claims

9AMENDED CLAIMS[received by the International Bureau on 2 June 1999 (02.06.99); original claims 1-4 amended; remaining claim unchanged (1 page)]
1. Bone-marrow derived myogenic precursor cells engineered with genes coding for dystrophin or distrophin-associated proteins.
2. Bone-marrow derived myogenic precursor cells according to claim 1 transduced with retroviral vector coding dystrophyn or associated proteins.
3. Bone-marrow derived myogenic precursor cells according to claim 1 coding for human dystrophyn, human utrophyn, human adalin, and variants thereof or functionally equivalent derivatives.
4. Bone-marrow derived myogenic precursor cells according to claim 1 coding for other proteins absent in other forms of muscular dystrophy.
5. Use of the cells according to anyone of claims 1-4 in the preparation of compositions for muscular dystrophy treatment.
PCT/EP1998/007263 1997-11-14 1998-11-13 Genetically-modified myogenic progenitors and their use in cell and gene therapy WO1999025814A1 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002310016A CA2310016A1 (en) 1997-11-14 1998-11-13 Genetically-modified myogenic progenitors and their use in cell and gene therapy
AU18725/99A AU1872599A (en) 1997-11-14 1998-11-13 Genetically-modified myogenic progenitors and their use in cell and gene therapy
JP2000521180A JP2001523448A (en) 1997-11-14 1998-11-13 Genetically modified myogenic precursors and their use in cells and gene therapy
EP98963450A EP1030908A1 (en) 1997-11-14 1998-11-13 Genetically-modified myogenic progenitors and their use in cell and gene therapy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ITMI97A002542 1997-11-14
IT97MI002542A IT1296439B1 (en) 1997-11-14 1997-11-14 GENETICALLY MODIFIED MYOGENIC PRECURSORS AND THEIR USE IN GENE AND CELL THERAPY

Publications (2)

Publication Number Publication Date
WO1999025814A1 WO1999025814A1 (en) 1999-05-27
WO1999025814B1 true WO1999025814B1 (en) 1999-07-22

Family

ID=11378206

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP1998/007263 WO1999025814A1 (en) 1997-11-14 1998-11-13 Genetically-modified myogenic progenitors and their use in cell and gene therapy

Country Status (6)

Country Link
EP (1) EP1030908A1 (en)
JP (1) JP2001523448A (en)
AU (1) AU1872599A (en)
CA (1) CA2310016A1 (en)
IT (1) IT1296439B1 (en)
WO (1) WO1999025814A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1216049A2 (en) * 1999-09-14 2002-06-26 Children's Medical Center Corporation Methods for treating muscular dystrophy with bone marrow cells
WO2005120526A1 (en) * 2004-06-11 2005-12-22 Combi Corporation Composition for inhibiting the progress of muscular dystrophy

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU7312891A (en) * 1990-02-12 1991-09-03 Board Of Regents, The University Of Texas System Satellite cell proliferation in adult skeletal muscle
US5591625A (en) * 1993-11-24 1997-01-07 Case Western Reserve University Transduced mesenchymal stem cells
GB9419048D0 (en) * 1994-09-20 1994-11-09 Watt Diana J Treatment of muscular disorders
EP0852463B1 (en) * 1995-06-06 2008-09-03 Case Western Reserve University Myogenic differentiation of human mesenchymal stem cells

Also Published As

Publication number Publication date
EP1030908A1 (en) 2000-08-30
WO1999025814A1 (en) 1999-05-27
AU1872599A (en) 1999-06-07
ITMI972542A1 (en) 1999-05-14
JP2001523448A (en) 2001-11-27
CA2310016A1 (en) 1999-05-27
IT1296439B1 (en) 1999-06-25

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