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Gene therapy, a booming therapeutic sector

Gene therapy offers the possibility of replacing the function of the defective or missing gene with that of a therapeutic gene inserted into the cell. The first diseases which benefited from gene therapy are rare genetic diseases in which the gene responsible had been identified, specifically severe immune deficiencies.

Illustration for article: What is gene therapy?

What is gene therapy?

Gene therapy consists of inserting a gene-drug into cells so that they can produce a protein to repair the anomaly responsible for the disease. This classic pathway, on which researchers have been working since the 1990s, has already produced its first results. Three of these world firsts were funded by AFM-Telethon through Telethon donations.
Illustration for article: The first successes in gene therapy

The first successes in gene therapy

Several world firsts were funded by the French Muscular Dystrophy Association (AFM-Téléthon) through Telethon donations, in particular the study which led to the first marketing authorisation application for gene therapy in 2009!