Contents
Gene therapy in children
Doctors first try to identify the affected gene (s). A very difficult step to take because many diseases are caused by many different genes (diabetes, hypertension, psychiatric illnesses, etc.).
Second step: we prepare the spare parts
To attack the evil at the root, the solution of gene therapy is to replace, at the heart of cells, and therefore in DNA, a diseased gene with its normal version. To prepare these “spare parts” to “enter” the cells, they “load” them into particular “carriers” or “vectors”: a virus (that of the common cold or even HIV for example), rendered completely harmless, or a “ball” of fat.
Third step: we repair
The drug genes are then administered, either directly into the organ to be treated or into the bloodstream, or onto cultured cells which will subsequently be reinjected into the patient. The healthy copy of the gene then takes over from the sick version and allows the body to regain normal functioning.
Gene therapy: a path strewn with pitfalls
This mode of cell repair is, in fact, very complicated at all stages. First of all that of the localization of the diseased gene which requires long-term studies on entire families; that of control of the vector, the action of the virus being only transitory, which sometimes necessitates repeating the operation on multiple occasions; that of the choice and quantity of genes; that of the possible rejection reaction, finally. Hence the few conclusive results obtained so far by the numerous trials in progress in the world, on animals, but also on humans. The areas of research are diseases of the nervous system (Parkinson’s or Alzheimer’s disease), of the heart, of the eye, of the skin, in the treatment of diabetes, cancer, and even obesity.
Progress on gene therapy
Following the success, in 2000, of the team of Prof. Alain Fisher, from the Necker hospital, in Paris, in the treatment of babies suffering from an extremely serious immunodeficiency linked to the X chromosome, the technique has progressed slowly. And this year, she even made great leaps forward. Indeed, in Italy, a team from Milan, supported by the French and Italian Telethons, is preparing to apply for a Marketing Authorization (AMM) for a gene therapy which makes it possible to treat children affected by an immune deficiency. particular. In France, Nathalie Cartier Lacave and Patrice Aubourg (Saint-Vincent de Paul and Inserm hospital) presented the first results of the trial they carried out successfully in two children aged 7 years suffering from adrenoleukodystrophy (ALD) linked at the X.