Wspólnotowy Serwis Informacyjny Badan i Rozwoju - CORDIS

FP6

POLEXGENE — Wynik w skrócie

Project ID: 19114
Źródło dofinansowania: FP6-LIFESCIHEALTH
Kraj: Belgium

Improving ex vivo gene therapy approaches

Leading scientists optimised non-viral gene delivery for ocular and cardiovascular ex vivo gene therapy.
Improving ex vivo gene therapy approaches
Gene therapy is an emerging technology for treating many hereditary genetic disorders by replacing the 'defective' gene in the target tissue. The gene can be delivered locally or by transplantation of corrected cells (ex vivo gene therapy). Despite the extensive research on the development of viral and non-viral vector systems, various side effects, including toxicity and immunogenicity, have hampered the efficacy of gene therapy.

The objective of the EU-funded 'Biocompatible non-viral polymeric gene delivery systems for the ex vivo treatment of ocular and cardiovascular diseases with high unmet medical need' (Polexgene) project was to develop a non-viral approach for ocular and cardiovascular disease. Project partners utilised polymeric compounds with a positive charge to complex the negatively charged DNA and enable its cellular uptake.

To enhance the DNA complex interaction with the cell, they incorporated cell-interacting peptides (CIP), an emerging technology in the field of gene delivery. CIP proteins are capable of translocation across the cell membrane carrying along the attached DNA to the inside of the cell.

The consortium used various techniques, including a novel impedance chip, to optimise the DNA polyplex formation and the efficiency of cellular uptake. Detailed chemical characterisation of the DNA polyplexes, analysis of the polyplex-cell interactions and subsequent biological evaluation of the efficiency of gene delivery in a variety of cell types were carried out. Study results showed that some novel polymers could deliver DNA in a more efficient and less toxic manner than previously used compounds.

Overall, the Polexgene project used CIP proteins to optimise and develop a novel non-viral gene delivery approach. Although pre-clinical evaluation will determine the in vivo efficacy of this approach, the findings of this study indicated that non-viral gene delivery with CIP proteins holds great promise as a safer alternative to current methods. Continuous research in the field will help improve the gene therapy technology and its broad application.

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