[go: up one dir, main page]

WO2008136670A3 - Improved methods and means for lentiviral gene delivery - Google Patents

Improved methods and means for lentiviral gene delivery Download PDF

Info

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
French (fr)
Other versions
WO2008136670A2 (en
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/en
Application filed by Erasmus University Medical Center filed Critical Erasmus University Medical Center
Publication of WO2008136670A2 publication Critical patent/WO2008136670A2/en
Publication of WO2008136670A3 publication Critical patent/WO2008136670A3/en
Anticipated expiration legal-status Critical
Ceased 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
    • 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

The invention relates to the field of gene therapy and more in specific to lentiviral gene delivery vehicles and methods for efficient transduction of lentiviral gene delivery vehicles into hematopoietic stem cells and their descendants. Preferably, the invention provides in one of its embodiments a method of gene transfer into e.g. pluripotent hematopoietic stem cells and their descendants, enabling successful transduction of cells, including transplantable cell populations comprising hematopoietic stem cells that give rise to progeny expressing the transduced gene(s). The invention further comprises a method for treating a variety of hereditary and acquired human disease by transfer of therapeutically active genes into hematopoietic stem cells. As a non-limiting example, the invention shows that symptoms associated with Pompe disease are (completely) reduced and/or alleviated by treatment of a subject suffering from Pompe disease withJiematopoietic stem cell transduced with an alpha- glucosidase comprising lentiviral vector.
PCT/NL2008/050269 2007-05-02 2008-05-02 Improved methods and means for lentiviral gene delivery Ceased WO2008136670A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
PCT/NL2007/050193 WO2008136656A1 (en) 2007-05-02 2007-05-02 Improved methods and means for lentiviral gene delivery
NLPCT/NL2007/050193 2007-05-02
EP07118404.8 2007-10-12
EP07118404 2007-10-12

Publications (2)

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

Family

ID=39944128

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/NL2008/050269 Ceased WO2008136670A2 (en) 2007-05-02 2008-05-02 Improved methods and means for lentiviral gene delivery

Country Status (1)

Country Link
WO (1) WO2008136670A2 (en)

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 (en) 2013-10-24 2015-04-30 Ospedale San Raffaele S.R.L. Method for the genetic modification of haematopoietic stem and progenitor cells (hspcs) and the use of genetically modified hspcs in gene therapy
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 (en) 2008-11-13

Similar Documents

Publication Publication Date Title
WO2008136670A3 (en) Improved methods and means for lentiviral gene delivery
Gehring Genomic imprinting: insights from plants
CA3136735A1 (en) Methods and compositions for editing rnas
KR20210053898A (en) New CRISPR enzyme and system
WO2010045002A3 (en) Gene therapy vectors and cytosine deaminases
WO2007130604A3 (en) Anti-tumor activity of an oncolytic adenovirus-delivered oncogene sirna
KR20200064168A (en) Systems, methods, and compositions for targeted nucleic acid editing
KR20190129088A (en) New CAS13B Ortholog CRISPR Enzyme and System
GB2472964A (en) Vectors for delivery of light-sensitive proteins and methods of use
GEP201606544B (en) Excision of transgenes in genetically modified organisms
JP2010530239A5 (en)
WO2008156702A3 (en) Bacteria mediated gene silencing
EP2851422A3 (en) CD34neg stem cells for the treatment of gastrointestinal disorders
WO2009135165A3 (en) Small humanin-like peptides
WO2012058522A3 (en) Cancer imaging with therapy: theranostics
WO2006018836A3 (en) Nucleic acid constructs and cells, and methods utilizing same for modifying the electrophysiological function of excitable tissues
US9107934B2 (en) MicroRNA as a cancer progression predictor and its use for treating cancer
WO2013096958A8 (en) Compositions and methods for the delivery of biologically active rnas
WO2010089122A3 (en) Vector(s) containing an inducible gene encoding a cdk4/cdk6 inhibitor useful for treating neurodegenerative disorders or diseases associated with an unscheduled activation of the cell cycle
WO2011028880A3 (en) Methods for enhancing genome stability and telomere elongation in embryonic stem cells
WO2007022030A3 (en) Methods and compositions for the efficient delivery of therapeutic agents to cells and animals
WO2006083797A3 (en) Improving and protecting cell therapy for neurological disorders including parkinson's disease
IN2014DN03383A (en)
Broughton et al. Myocardial Regeneration for Humans―Modifying Biology and Manipulating Evolution―
WO2006053245A3 (en) Retroviral vectors with introns

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 08741690

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 08741690

Country of ref document: EP

Kind code of ref document: A2