Gene therapy is created through the use of a mobile DNA sequence


Gene therapy without using viruses was developed by a team of researchers at the University of Leuven, the VIB (Flanders Institute of Biotechnology) and the Max Delbrück Center Berlin. They climbed a major step to implement it one day to patients by developing a safe genetic transfer "vehicle" . Gene therapy involves the introduction of genes into cells or tissues of an individual to treat a disease. This process has seen significant progress in recent years. But for the genetic code within the cell to correct, scholars have until now used a virus.

This attenuated virus, modified to avoid transmission of diseases. Dr. Thierry Vandendriessche of VIB emphasizes that there is still a risk of side effects. He explained in some cases, the virus causes inflammation and even cancer. The adenovirus, for example, infects two-thirds of the population, without significant health effects. But if it is used to carry "gene correction" to someone who has developed antibodies to this virus, you can create a serious immune problem. To avoid these side effects, a solution was found. It is a manipulation to make a transfer using a non-viral vector. To do this, the researchers used a transposon "transposable genetic element".

Transposons, generally known for their importance in the transfer of functions, are mobile elements consisting of a sequence of DNA. They are likely to change their location on the chromosome. SB for "sleeping beauty" transposon is chosen by the team of Leuven among thousands of candidates. It is derived from a fish transposon, also active in mice, rats and frogs. The transfer was made to CD34 + cells, cells that are found in stem cells from bone marrow and umbilical cord blood, which is precisely the peculiarity of being both very difficult to "transfer" and very useful to treat due to their ability to transform into different cells of the body.

After implanted these cells in vivo in mice, researchers have observed no side effects in this group. Gene transfer correction was therefore perfectly fulfilled and sustained (more than 5 months). The selection and modification of complex transposon original experience which has resulted in a success. Gene therapy without using viruses will be more reliable to treat a disease. Note that this is the first, the use of a transposable genetic element, transposon, to bring about corrective genes in the heart of the cell.

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