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INTHER — Result In Brief

Project ID: 18961
Country: Germany

Improving on gene therapy applications

European scientists strengthened the application of gene therapy by developing novel gene delivery strategies based on mobile genetic elements.
Improving on gene therapy applications
Gene therapy is the method of correcting defective genes in one's cells. The ex vivo gene therapy applications endow removal of a patient's cells, introduction of the therapeutic gene and re-implantation of the cells into the patient. Delivery of the therapeutic gene usually occurs by viral or non-viral constructs. However, safety concerns and efficiency limitations of current systems necessitate the development of novel gene transfer technologies.

.The objective of the EU-funded 'Development and application of transposons and site-specific integration technologies as non-viral gene delivery methods for ex vivo gene-based therapies' (Inther) project was to develop gene transfer vectors based on transposons. Transposons are DNA sequences capable of 'jumping' around in the host genome. This unique feature can be exploited to mediate targeted integration of a desired gene in the cell genome.

.The consortium constructed transposon vectors and transferred them to cells ex vivo using non-viral DNA delivery methods. The sites in the genome where these vectors had integrated were explored to address safety issues of transposon vector administration. Team members also tested these vectors in vivo in animal models of various diseases including copper metabolism disease, anaemia, chronic granulomatous disease, bleeding and neurological disorders.

Gene therapy is a rapidly expanding technology but safety concerns limit its application. The Inther project thus advanced the possibilities for alternative tools to current viral and non-viral technologies for somatic gene therapy.

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