Parkinson disease is a neurodegenerative pathology with high incidence. Current treatments ease the symptoms but don't stop the development of the disease and aren't without any major side effects. Although this pathology is not specifically caused by genetic abnormalities, the involvement of numerous proteins in the pathophysiological process enables us to give an interest to gene therapy. This hypothesis has been upheld by positive results on animals and by five phase I trials on humans but may be attenuated by the first phase II trial recently published showing modest efficiency and multiple side effects. However, these preliminary results will need to be reinforced by more important trials in order to be sure of the safety and get efficacy data, which will allow us to give an opinion upon this new way of treatment.
© 2011 Société Française de Pharmacologie et de Thérapeutique.