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WO2008136670A3 - Procédés et moyens améliorés pour une distribution de gène lentiviral - Google Patents

Procédés et moyens améliorés pour une distribution de gène lentiviral Download PDF

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Publication number
WO2008136670A3
WO2008136670A3 PCT/NL2008/050269 NL2008050269W WO2008136670A3 WO 2008136670 A3 WO2008136670 A3 WO 2008136670A3 NL 2008050269 W NL2008050269 W NL 2008050269W WO 2008136670 A3 WO2008136670 A3 WO 2008136670A3
Authority
WO
WIPO (PCT)
Prior art keywords
stem cells
hematopoietic stem
gene
gene delivery
lentiviral
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.)
Ceased
Application number
PCT/NL2008/050269
Other languages
English (en)
Other versions
WO2008136670A2 (fr
Inventor
Til Nico Peter Van
Monique Maria Andrea Verstegen
Gerard Wagemaker
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.)
Erasmus University Medical Center
Original Assignee
Erasmus University Medical Center
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
Priority claimed from PCT/NL2007/050193 external-priority patent/WO2008136656A1/fr
Application filed by Erasmus University Medical Center filed Critical Erasmus University Medical Center
Publication of WO2008136670A2 publication Critical patent/WO2008136670A2/fr
Publication of WO2008136670A3 publication Critical patent/WO2008136670A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

L'invention porte sur le domaine de la thérapie génique et plus spécifiquement sur des véhicules de distribution de gène lentiviral et sur des procédés pour une transduction efficace de véhicules de distribution de gène lentiviral dans des cellules souches hématopoïétiques et leurs descendants. De préférence, l'invention concerne dans l'un de ces modes de réalisation un procédé de transfert de gène dans, par exemple, des cellules souches hématopoïétiques pluripotentes et leurs descendants, permettant une transduction réussie des cellules, comprenant des populations cellulaires transplantables comprenant des cellules souches hématopoïétiques, qui donnent lieu à une descendance exprimant le ou les gènes transduits. L'invention comprend en outre un procédé pour traiter une diversité de maladies humaines héréditaires et acquises par transfert de gènes thérapeutiquement actifs dans les cellules souches hématopoïétiques. En tant qu'exemple non limitatif, l'invention montre que des symptômes associés à la maladie de Pompe sont (entièrement) réduits et/ou soulagés par traitement d'un sujet souffrant de la maladie de Pompe avec une cellule souche hématopoïétique transduite par un vecteur lentiviral comprenant une alpha-glucosidase.
PCT/NL2008/050269 2007-05-02 2008-05-02 Procédés et moyens améliorés pour une distribution de gène lentiviral Ceased WO2008136670A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
PCT/NL2007/050193 WO2008136656A1 (fr) 2007-05-02 2007-05-02 Procédés perfectionnés et moyens pour l'administration de gènes lentiviraux
NLPCT/NL2007/050193 2007-05-02
EP07118404.8 2007-10-12
EP07118404 2007-10-12

Publications (2)

Publication Number Publication Date
WO2008136670A2 WO2008136670A2 (fr) 2008-11-13
WO2008136670A3 true WO2008136670A3 (fr) 2009-02-19

Family

ID=39944128

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/NL2008/050269 Ceased WO2008136670A2 (fr) 2007-05-02 2008-05-02 Procédés et moyens améliorés pour une distribution de gène lentiviral

Country Status (1)

Country Link
WO (1) WO2008136670A2 (fr)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11286501B2 (en) 2016-04-20 2022-03-29 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas O.A, M.P. Methods of treating or preventing pyruvate kinase deficiency
US11642422B2 (en) 2017-10-16 2023-05-09 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A, M.P. Lentiviral vectors for delivery of PKLR to treat pyruvate kinase deficiency

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2927712A1 (fr) 2013-10-24 2015-04-30 Ospedale San Raffaele S.R.L. Methode de modification genetique des cellules souches et precurseurs hematopoietiques (hspcs) et utilisation des hspcs genetiquement modifies dans la therapie genique
NL2019517B1 (en) * 2017-09-08 2019-03-19 Univ Erasmus Med Ct Rotterdam New therapy for Pompe disease
AU2021202658A1 (en) * 2021-04-28 2022-11-17 Fondazione Telethon Gene therapy
NL2031676B1 (en) 2022-04-22 2023-11-07 Univ Erasmus Med Ct Rotterdam Gene therapy for Pompe Disease
AU2023372338A1 (en) 2022-11-01 2025-05-22 Erasmus University Medical Center Rotterdam Gene therapy constructs for metabolic disorders

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BRENNER SEBASTIAN ET AL: "The late dividing population of gamma-retroviral vector transduced human mobilized peripheral blood progenitor cells contributes most to gene-marked cell engraftment in nonobese diabetic/severe combined immunodeficient mice.", STEM CELLS (DAYTON, OHIO) JUL 2007, vol. 25, no. 7, July 2007 (2007-07-01), pages 1807 - 1813, XP009095635, ISSN: 1066-5099 *
SU LISHAN ET AL: "Hematopoietic stem cell-based gene therapy for acquired immunodeficiency syndrome: Efficient transduction and expression of RevM10 in myeloid cells in vivo and in vitro", BLOOD, vol. 89, no. 7, 1997, pages 2283 - 2290, XP002468028, ISSN: 0006-4971 *
SUTTON R E ET AL: "TRANSDUCTION OF HUMAN PROGENITOR HEMATOPOIETIC STEM CELLS BY HUMAN IMMUNODEFICIENCY VIRUS TYPE 1-BASED VECTORS IS CELL CYCLE DEPENDENT", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, vol. 73, no. 5, May 1999 (1999-05-01), pages 3649 - 3660, XP002937676, ISSN: 0022-538X *
VAN TIL N ET AL: "Cell density and viral vector load are determinant factors for ex vivo lentiviral transduction effeciency of hematopoietic stem cells", AMERICAN SOCIETY OF GENE THERAPY ANNUAL MEETING, XX, XX, 30 May 2007 (2007-05-30) - 3 June 2007 (2007-06-03), XX, pages 118, XP002468030 *
ZHANG B ET AL: "The significance of controlled conditions in lentiviral vector titration and in the use of multiplicity of infection (MOI) for predicting gene transfer events", GENETIC VACCINES AND THERAPY 04 AUG 2004 UNITED KINGDOM, vol. 2, 4 August 2004 (2004-08-04), pages 10p, XP002468027, ISSN: 1479-0556 1479-0556 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11286501B2 (en) 2016-04-20 2022-03-29 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas O.A, M.P. Methods of treating or preventing pyruvate kinase deficiency
US11642422B2 (en) 2017-10-16 2023-05-09 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A, M.P. Lentiviral vectors for delivery of PKLR to treat pyruvate kinase deficiency

Also Published As

Publication number Publication date
WO2008136670A2 (fr) 2008-11-13

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