Gene technology based therapies in the brain.

Abstract

Gene therapy potentially represents one of the most important developments in modern medicine. Gene therapy, especially of cancer, has created exciting and elusive areas of therapeutic research in the past decade. In fact, the first gene therapy performed in a human was not against cancer but was performed to a 14 year old child suffering from adenosine deaminase (ADA) deficiency. In addition to cancer gene therapy there are many other diseases and disorders where gene therapy holds exciting and promising opportunities. These include amongst others gene therapy within the central nervous system and the cardiovascular system. Improvements of the efficiency and safety of gene therapy is the major goal of gene therapy development. After the death of Jesse Gelsinger, the first patient in whom death could be directly linked to the viral vector used for the treatment, ethical doubts were raised about the feasibility of gene therapy in humans. Therefore, the ability to direct gene transfer vectors to specific target cells is also a crucial task to be solved and will be important not only to achieve a therapeutic effect but also to limit potential adverse effects.