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

Objetivo

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.

Convocatoria de propuestas

Data not available

Régimen de financiación

DEM - Demonstration contracts

Coordinador

INSTITUTO DE BIOLOGIA EXPERIMENTAL E TECNOLOGICA
Aportación de la UE
Sin datos
Dirección
Quinta do Marques
2780-901 OEIRAS
Portugal

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Coste total
Sin datos

Participantes (4)