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Mobile DNA outsmarts viral vectors

Gene Therapy - Researchers have developed a new non-viral approach to gene therapy that they say will overcome side-effects associated with the current viral vectors - such as inflammation or the development of cancer.

The success of gene therapy ultimately depends on these gene delivery vehicles or vectors. Most vectors have been derived from viruses that can be tailor-made to deliver therapeutic genes into the patients' cells. However, some of these viral vectors can induce side effects, including cancer and inflammation.

Now a team from the Katholieke Universiteit Leuven in Belgium) ND the Max Delbrück Center in Germany now developed a new non-viral approach based on transposons. Transposons are mobile DNA elements that can integrate into 'foreign' DNA via a 'cut-and-paste' mechanism. The researchers constructed the transposons in such a way that they can carry the therapeutic gene into the target cell DNA. Doing so, they obviate the need to rely on viral vectors.

"We show for the first time that it is now possible to efficiently deliver genes into stem cells, particularly those of the immune system, using non-viral gene delivery," said member of the team Marinee Chuah. "Many groups have tried this for many years but without success. We are glad that we could now overcome this hurdle"

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