Final Report Summary - INTHER (Development and application of transposons and site-specific integration technologies as non-viral gene delivery methods for ex vivo gene-based therapies)
Therapeutic gene constructs were made in transposon vectors, and delivered into cells ex vivo, using cutting edge non-viral nucleic acid delivery methods. Application of reversible implantation systems (collagen implants, skin biopumps, and encapsulated cells) were also explored. Animal disease models included copper metabolism diseases, anemia, hypercholesterolemia, bleeding disorders, chronic grnulomatous disease and neurological disorders. Genomic insertion, transgene expression and phenotypic correction were also investigated. Safety issues of transposon vector administration and genomic integration were addressed. The involvement of three small and medium-sized enterprises (SMEs) mobilised industrial know-how and technology. This project developed, tested and applied new therapeutic tools for somatic gene therapy as an alternative to currently used viral and non-viral technologies, and have the potential to offer new solutions for diseases that impose significant impairment to citizens' quality of life, as well as burdens on health care services in Europe.