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Integrated strategy for efficient therapeutic retrovirus production based on modular cell lines

Objective

To increase successful applications of retrovirus delivery in gene therapy this demonstration project integrates and validates novel but robust technologies:
i) Specific genetically stable and safer human diploid cells yield higher virus titers than established, transformed cell lines;
ii) Modular master producer cell lines permit predictable expression properties of the therapeutic construct and envelope genes, inserted precisely at defined, optimal loci, conserving expression levels, physiological, genetic stability and safety advantages of the master cells;
iii)Medium, cultivation, purification and storage improvements result in maximal cell and tissue infectivity;
iv) Safety and efficacy of cells and retroviral vectors will be thoroughly tested and certified, including examination in solid tissue organ systems;
v) A final cGMP run will validate and complete the integrated strategy to produce clinically applicable material for trials against dystrophic epidermolysis bullosa.

Call for proposal

Data not available

Coordinator

INSTITUTO DE BIOLOGIA EXPERIMENTAL E TECNOLOGICA
Address
Quinta Do Marques
2780-901 Oeiras
Portugal
 

Participants (4)

GBF - NATIONAL CENTRE FOR BIOTECHNOLOGY
Germany
Address
Mascheroder Weg 1
38124 Braunschweig
 
GENETHON III
France
Address
Rue De L'internationale 1Bis
91002 Evry
 
HENOGEN S.A.
Belgium
Address
Rue Des Professeurs Jaener Et Brachet 12
6041 Gosselies
 
THE HEBREW UNIVERSITY OF JERUSALEM - THE AUTHORITY FOR RESEARCH AND DEVELOPMENT
Israel
Address
Givat Ram Campus, Edmond Safra
91904 Jerusalem