Gene therapy - ISS (EN)

Advanced therapies are based on the use of genes, tissues or cells. Gene therapy medicines use recombinant genes to achieve a therapeutic, diagnostic or disease prevention effect. The recombinant genes will or will not be integrated into the host genome.

Gene therapy is used, for example, to supplement adefective biological product resulting from a mutated gene responsible for a disease, to inactivate a malfunctioning gene or to introduce new genes with the aim of counteracting a specific pathology.

Genes are administered through a vector, a vehicle used to make the gene penetrate into the target cells. Vectors can be administered to the patient directly or after inserting them into explanted cells which are then re-administered.