Gene therapy – from idea to reality.

Gene therapy - from idea to reality Gene therapy was originally proposed 45 years ago, but it is only during the last 5-10 years that significant clinical benefit has been demonstrated. Gene therapy is in most cases in the form of engineered viruses carrying a therapeutic gene. Examples of successfully treated disorders are primary immunodeficiencies and hemophilias. In some cases, gene therapy consists of genetically modified cells, such as when chimeric antigen receptors are stably introduced into T lymphocytes, and used as tumor therapy, mainly for leukemias. Genetic therapy also includes oligonucleotides, which consist of around 20 nucleotides. Several such compounds have been approved for clinical use. Gene editing, which was a utopia, only a few years ago, has now become a clinical reality. In the main, rather small patient groups have been treated and a future challenge is the scale-up of manufacturing processes and the cost-effective use of the new therapies.