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 a defective 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.