ProSavin

ProSavin is an experimental gene therapy product developed for the treatment of Parkinson's disease. It aims to deliver three genes (TH, AADC, and CH1) directly into the putamen region of the brain. These genes encode enzymes that are necessary for the production of dopamine, a neurotransmitter that is deficient in individuals with Parkinson's disease. The therapy uses a lentiviral vector to deliver the genes, enabling them to integrate into the patient's DNA and potentially provide long-term dopamine production. Clinical trials have been conducted to evaluate the safety and efficacy of ProSavin in patients with advanced Parkinson's disease. While early trials showed some promise, further development and trials are necessary to determine its long-term effectiveness and role in Parkinson's disease treatment. The mechanism seeks to compensate for the loss of dopamine-producing cells, a hallmark of the disease.