Gene therapy is an exciting technique which aims to treat or prevent diseases through the introduction of genes into patient’s cells. Typically, gene therapy works by inserting a working copy of a gene into cells to replace the function of a faulty copy which is causing a disease- this might be because the faulty gene is producing a defective form of a critical protein. It can also be utilized to insert a completely new gene to make a beneficial protein in cells. Therefore unlike existing pharmaceuticals, gene therapy aims to treat the cause of genetic diseases and not just the symptoms.
Whilst gene therapy is concerned with the delivery of DNA into cells, cell therapy involves the transfer of cells into a patient’s body with the aim of treating a disease. These cells may be derived from a donor or taken from and reinfused into the same individual.