Gene therapy: into the future of medicine
Jonas Araújo de Souza discusses different approaches and the relevance to modern medicine
The sequencing of the human genome has been compared to putting a man on the moon, and it will certainly change health care, but the most important work lies ahead, in determining how to put the information to medical use. In this context, applications such as gene therapy are being explored. What was once seen as a science fiction dream is now becoming a real possibility.
Gene therapy is a new form of drug delivery that leads the patient's own cells to produce a therapeutic agent.12 It could potentially eliminate the need for repeated administration of proteins or drugs. Applications of gene therapy not only include rare inherited diseases but extend to common acquired disorders, including tumours (predominantly malignant melanoma) and haematological disorders, cardiovascular disease, and the acquired immunodeficiency syndrome (fig 1). Gene therapy therefore could be a key element of medical practice in the future.
Gene therapy was tried