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 proposalData not available
Funding SchemeDEM - Demonstration contracts