Developing innovative therapies
Correcting a defective gene, inserting a gene-drug, grafting cells capable of regenerating healthy tissue: AFM-Téléthon paves the way for the medicine of the future and supports about 37 clinical trials.
Gene surgery : repairing the gene
Gene surgery involves repairing the gene itself. Exon skipping, stop codon read-through, meganucleases: these techniques are less than ten years old but already undergoing human trials!
Cell therapy : replacing the deficient cells
Cell therapy takes up the challenge of replacing deficient or absent cells with healthy cells. It succeeds mainly through the contribution of specific cells: stem cells and iPS. From cardiac insufficiency to neuromuscular disease via cancer or nervous system pathologies, there are numerous applications.
Drugs: a traditional pathway
New molecules are constantly being discovered and existing drugs have not yet reached their full potential. AFM-Téléthon supports research into new molecules as well as existing pharmacological treatments.
Ongoing trials and projects
Every year, AFM-Téléthon supports clinical trials designed to evaluate the efficacy of innovative treatments. Using Telethon donations, it supports more than 30 clinical trials for 29 different diseases.
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 were rare genetic diseases in which the gene responsible had been identified, severe immune deficiencies.