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Combined isolation and stable non-viral transfection of hematopoietic cells- a novel platform technology for ex vivo hematopoietic stem cell gene therapy

Combined isolation and stable non-viral transfection of hematopoietic cells- a novel platform technology for ex vivo hematopoietic stem cell gene therapy

Objective

The feasibility of ex vivo gene therapy in humans has been demonstrated with retrovirally ransduced hematopoietic stem cells. At the same time, risks associated with the use of retroviral vectors became apparent. We will establish and validate a novel combination technology for hematopoietic cell isolation and non-viral transfection leading to site-specific genomic integration of transfected nucleic acids. This novel technology will be validated by expert groups in hematopoietic stem cell gene therapy and related basic and clinical research. The technology is going to be exploited and disseminated by the company partners Miltenyi Biotech, OZ Biosciences, and Poetic Genetics.

A platform technology for non-viral transfection of hematopoietic cells in general and hemaptopoietic stem cells in particular, with optional stable genetic modification, will be established by integrating a clinically approved magnetic cell separation technique (CliniMACS) with magnetically enhanced transfection (Magnetofection). Cel l nucleus-targeting vectors will be applied. For selected indications, the technology will be practiced with plasmid constructs that provide site-specific genomic integration, either the phage phiC31 integrase system or, alternatively, a drug-inducible A AV-derived replicase/integrase system.

Technology validation includes the analysis of genomic integration sites, transcriptom profiling, characterization of stable and inducible trans-gene expression and evaluation of engraftment and persistence in transgenic animal models using molecular biological tools and magnetic resonance imaging. The therapeutic potential will be examined in a SCID-X mouse model in direct comparison with established retroviral technology. Magselectofection is expected to circumvent problems associated with viral vectors, contribute to health care progress and foster the competitiveness of Europe's biotechnology industry based on its competitive advantage.

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Coordinator

KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIVERSITÄT MÜNCHEN

Address

Ismaninger Str. 22
Munich

Germany

Administrative Contact

Participants (10)

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COMENIUS UNIVERSITY

Slovakia

ERASMUS UNIVERSITY MEDICAL CENTER

Netherlands

FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR

Italy

FRAUNHOFER GESELLSCHAFT ZUR FÖRDERUNG DER ANGEWANDTEN FORSCHUNG E.V.

Germany

LUDWIG-MAXIMILIANS-UNIVERSITÄT

Germany

M. SKLODOWSKA-CURIE MEMORIAL CANCER CENTER AND INSTITUTE OF ONCOLOGY

Poland

MILTENYI BIOTEC GMBH

Germany

OZ BIOSCIENCES

France

RESEARCH INSTITUTE OF MOLECULAR PATHOLOGY

Austria

WEIZMANN INSTITUTE OF SCIENCE

Israel

Project information

Grant agreement ID: 19038

  • Start date

    1 May 2006

  • End date

    31 December 2009

Funded under:

FP6-LIFESCIHEALTH

  • Overall budget:

    € 3 317 000

  • EU contribution

    € 2 800 000

Coordinated by:

KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIVERSITÄT MÜNCHEN

Germany