WO1996021733A3 - Transfert of dna in cells with the view to correcting the adrenoleucodystrophy - Google Patents

Transfert of dna in cells with the view to correcting the adrenoleucodystrophy Download PDF

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Publication number
WO1996021733A3
WO1996021733A3 PCT/FR1996/000059 FR9600059W WO9621733A3 WO 1996021733 A3 WO1996021733 A3 WO 1996021733A3 FR 9600059 W FR9600059 W FR 9600059W WO 9621733 A3 WO9621733 A3 WO 9621733A3
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WO
WIPO (PCT)
Prior art keywords
adrenoleucodystrophy
vector
transfert
correcting
dna
Prior art date
Application number
PCT/FR1996/000059
Other languages
French (fr)
Other versions
WO1996021733A2 (en
WO1996021733A9 (en
Inventor
Jean-Louis Mandel
Patrick Aubourg
Jean Mosser
Claude-Olivier Sarde
Original Assignee
Inst Nat Sante Rech Med
Mandel Jean Louis
Patrick Aubourg
Jean Mosser
Sarde Claude Olivier
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 Inst Nat Sante Rech Med, Mandel Jean Louis, Patrick Aubourg, Jean Mosser, Sarde Claude Olivier filed Critical Inst Nat Sante Rech Med
Priority to AU45431/96A priority Critical patent/AU4543196A/en
Publication of WO1996021733A2 publication Critical patent/WO1996021733A2/en
Publication of WO1996021733A3 publication Critical patent/WO1996021733A3/en
Publication of WO1996021733A9 publication Critical patent/WO1996021733A9/en

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    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention relates to a recombinant vector usuable to introduce a nucleic acid sequence into a target cell, characterized in that it comprises: a nucleotide sequence derived from a cDNA on the gene whose mutuation is responsible for the adrenoleucodystrophy or the adrenomyelopathy and which codes for a membrane protein; a retroviral vector M48 wherein said nucleotide sequence is inserted. According to an another embodiment of the invention, the vector M48 is replaced by another retroviral, viral or chemical vector.
PCT/FR1996/000059 1995-01-13 1996-01-12 Transfert of dna in cells with the view to correcting the adrenoleucodystrophy WO1996021733A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU45431/96A AU4543196A (en) 1995-01-13 1996-01-12 Transfert of dna in cells with the view to correcting the adrenoleucodystrophy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
FR9500376A FR2729399B1 (en) 1995-01-13 1995-01-13 RECOMBINANT RETROVIRAL VECTOR FOR CORRECTION OF GENE TRANSFER ADRENOLEUCODYSTROPHY
FR95/00376 1995-01-13

Publications (3)

Publication Number Publication Date
WO1996021733A2 WO1996021733A2 (en) 1996-07-18
WO1996021733A3 true WO1996021733A3 (en) 1996-09-12
WO1996021733A9 WO1996021733A9 (en) 1996-11-21

Family

ID=9475123

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/FR1996/000059 WO1996021733A2 (en) 1995-01-13 1996-01-12 Transfert of dna in cells with the view to correcting the adrenoleucodystrophy

Country Status (3)

Country Link
AU (1) AU4543196A (en)
FR (1) FR2729399B1 (en)
WO (1) WO1996021733A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2775694B1 (en) * 1998-03-06 2000-06-09 Gradient Ass TOXICITY TEST
US20050163760A1 (en) * 2001-12-06 2005-07-28 Nathalie Cartier-Lacave Use of cd34+ hematopoietic progenitor cells for the treatment of cns disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994024298A1 (en) * 1993-04-21 1994-10-27 Institut Pasteur Biocompatible implant for the expression and secretion in vivo of a therapeutical compound
CA2108606A1 (en) * 1993-10-15 1995-04-16 Jean-Louis Mandel X-linked adrenoleukodystrophy gene and corresponding protein

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994024298A1 (en) * 1993-04-21 1994-10-27 Institut Pasteur Biocompatible implant for the expression and secretion in vivo of a therapeutical compound
CA2108606A1 (en) * 1993-10-15 1995-04-16 Jean-Louis Mandel X-linked adrenoleukodystrophy gene and corresponding protein

Non-Patent Citations (13)

* Cited by examiner, † Cited by third party
Title
B.C. GUILD ET AL.: "Development of retrovirus vectors useful for expressing genes in cultured murine embryonal cells and hematopoietic cells in vivo", J. VIROLOGY, vol. 62, no. 10-10, October 1988 (1988-10-01), AM.SOC.MICROBIOL.,WASHINGTON,US, pages 3795 - 3801, XP002005766 *
CARTIER, NATHALIE ET AL: "Retroviral-mediated gene transfer corrects very-long-chain fatty acid metabolism in adrenoleukodystrophy fibroblasts", PROC. NATL. ACAD. SCI. U. S. A. (1995), 92(5), 1674-8 CODEN: PNASA6;ISSN: 0027-8424, 28 February 1995 (1995-02-28), XP002005770 *
H. W. MOSER ET AL.: "gene therapy in neurology", EUROPEAN NEUROLOGY, vol. 34, no. 5, October 1994 (1994-10-01), KARGER, BASEL, CH, pages 241 - 242, XP002005760 *
J. MOSSER ET AL.: "Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters", NATURE, vol. 361, 25 February 1993 (1993-02-25), MACMILLAN JOURNALS LTD., LONDON,UK, pages 726 - 730, XP002005763 *
J. MOSSER ET AL.: "The gene responsable for adrenoleukodystrophy encodes a peroxisomal membrane protein", HUMAN MOLECULAR GENETICS, vol. 3, no. 2, February 1994 (1994-02-01), OXFORD UNIVERSITY PRESS, UK, pages 265 - 271, XP002005762 *
L. TENNENBAUM ET AL.: "Adeno-associated virus (AAV) as a vector for gene transfer into glial cells of the human central nervous system", GENE THERAPY, vol. 1, no. s1, 1993, pages S80, XP002005769 *
L.LU ET AL.: "High efficiency retroviral mediated gene transduction into single isolated immature and replatable CD34+ hematopoietic stem/progenitor cells from human umbilical cord blood", J. EXP. MED., vol. 178, no. 6, December 1993 (1993-12-01), ROCKEFELLER UNIV. PRESS, US, pages 2089 - 2096, XP002005684 *
M.G. KAPLITT ET AL.: "Adenovirus-associated virus (AAV) vectors for genetic therapy of neurological disease", NEUROLOGY VOL. 44 (4 SUUPL. 2), 1994, pages A163, XP002005768 *
M.G. KAPLITT ET AL.: "Long-term gene expression and phenotypic correction using adeno-associated virus vector in the mammalian brain", NATURE GENETICS, vol. 8, October 1994 (1994-10-01), NATURE PUBLISHING CO., NEW YORK, US, pages 148 - 154, XP002005767 *
P. AUBOURG ET AL.: "Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation", THE NEW ENGLAND JOURNAL OF MEDCINE, vol. 322, no. 26, 28 June 1990 (1990-06-28), MA. MED. SOC., BOSTON, MA,US, pages 1860 - 1866, XP002005765 *
SHINNOH N ET AL: "Adrenoleukodystrophy: The restoration of peroxisomal beta-oxidation by transfection of normal cDNA.", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS 210 (3). 1995. 830-836. ISSN: 0006-291X, 15 May 1995 (1995-05-15), XP002005771 *
T. FRIEDMANN: "Gene therapy for neurological disorders", TRENDS IN GENETICS, vol. 10, no. 6, June 1994 (1994-06-01), ELSEVIER SCIENCE LTD., AMSTERDAM, NL, pages 210 - 214, XP002005761 *
T. OHASHI ET AL.: "Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector", PROC. NATL.ACAD SCI., vol. 89, no. 23, 1 December 1992 (1992-12-01), NATL. ACAD SCI.,WASHINGTON,DC,US;, pages 11332 - 11336, XP002005764 *

Also Published As

Publication number Publication date
WO1996021733A2 (en) 1996-07-18
FR2729399A1 (en) 1996-07-19
FR2729399B1 (en) 1997-04-04
AU4543196A (en) 1996-07-31

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