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Aurora magazine

What is gene therapy?

Gene therapy is born with the primary purpose of addressing genetic diseases. In spite of this, applications in the last few years are flourishing applications such as infarction, tumors, Alzheimer's and Parkinson's. The merit of this success is the flexibility demonstrated by the technique, applicable wherever there is a defective gene to be repaired.

The technique consists in the removal of some defective cells from the patient, which are subsequently corrected and reintroduced into the body. For this purpose, scientists use a virus that is harmless to introduce proper DNA into cells. DNA heals treated cells, which once multiply in the body and replace the diseased ones. Beyond the theory, there are of course obstacles that make practice much more complex.

The first hits date back to 1990 when Claudio Bordignon used the technique on a sick child of Ada-Scid. Despite a first healing, the therapeutic effect of the cells disappeared at the end of their life cycle. Subsequently, the American French Anderson and Michael Blaise made the permanent effect by modifying bone marrow stem cells. Again this time, the results were only partial: the experiments of the new technique caused several deaths and stopped.

2009 was the year of the new beginning for gene therapy. A study by the Telethon Institute of Medical Genetics (Tigem) in Naples returned the sight to a child suffering from amaurosis of Leber. The merit was the discovery of a new vector for transporting DNA in the cell, safer than the previous ones. However, the virus used was not perfect, which required new experiments to find alternatives. Some of these are still under way.
A major breakthrough for gene therapy was the study of the team of Luigi Naldini in 2013. Nandini announced that it has cared for six children at risk of rare genetic diseases. Three had the metachromatic leukodystrophy gene and three others of Wiskott-Aldrich syndrome. Scientists ran stem cells and used them to replace those sick, blocking the development of the disease.

To date, the steps forward in gene therapy are many. Drugs are being developed for the treatment of tumors, leukemia and genetic diseases.

Source: focus. it