Perspectives of gene therapy

Abstract

The concept of gene therapy is really quite simple: delivery of genetic material to cells with the objective of impacting the progression of a disease. Even with this easy to understand definition, the field of gene therapy is actually rich in technologic innovations and interdisciplinary knowledge and practice. The development and application of gene therapy encounter significant challenges just as is seen with any experimental treatment, but also encounters additional difficulties that are particular to the use of biological material as a pharmacological agent. This short review will present a few basic concepts about the technology and application of gene therapy and will provide the basis for discussing the important role that is played by the association of basic and clinical researchers during the development of these new therapeutic approaches. The contribution of knowledge by specialists in the laboratory and clinical aspects permits the rapid progress of both technology and its application as gene therapy in patients. In Brazil, gene therapy is still at an early phase of development as compared to many countries around the world, yet our advances are significant and we have excellent perspectives for growth in this area.