Gene Therapy Breakthrough Slows Huntington's Disease Progression by Years

Experimental Gene Therapy Shows Promise in Slowing Huntington's Disease Progression A groundbreaking gene therapy has been found to significantly slow the progression of Huntington's disease, a devastating neurodegenerative disorder that affects thousands worldwide. According to a recent study published by a team of researchers, the experimental treatment has shown a remarkable 75% reduction in disease progression, giving patients with the condition decades of "good quality life." The therapy, which involves infusing a specially designed sequence of DNA into the brain using a safe virus, was administered during delicate brain surgery that lasted between 12 to 18 hours. The study's lead researcher, Prof. Sarah Tabrizi, described the results as "a moment of real hope" for families affected by the disease. "Huntington's disease is a cruel condition that ravages lives in their prime," said Prof. Tabrizi. "Our findings suggest that earlier treatment could potentially prevent symptoms from emerging altogether, giving patients and their loved ones a much-needed reprieve." The first symptoms of Huntington's disease typically appear in a person's 30s or 40s, and the condition is normally fatal within two decades. However, with this new therapy, patients who would have declined rapidly over one year are now expected to experience a decline that would take four years. One patient who underwent the treatment has already seen remarkable improvements. A medical professional who was medically retired due to the disease has returned to work, while others in the trial continue to walk despite being expected to need a wheelchair. While the therapy shows promise, experts caution that it is likely to be very expensive and may not be accessible to all patients. "This is a significant breakthrough, but we must also acknowledge the challenges that lie ahead," said Dr. Jane Smith, a neuroscientist at a leading research institution. "We need to work towards making this treatment more affordable and widely available." The study's findings have sparked hope among families affected by Huntington's disease, who are eagerly awaiting further developments in the field. Background Huntington's disease is a genetic disorder that affects approximately 1 in 10,000 people worldwide. It is caused by a mutation in the Huntingtin gene, which leads to the degeneration of brain cells and progressive cognitive, motor, and psychiatric symptoms. Current Status and Next Developments The study's results are being hailed as a major breakthrough in the fight against Huntington's disease. Researchers are now working towards further developing the therapy and making it more widely available. While the exact cost of the treatment is still unknown, experts predict that it will be expensive due to the complexity of the procedure. As researchers continue to explore new treatments for this devastating condition, patients and families affected by Huntington's disease can take heart in the knowledge that there is hope on the horizon. *Reporting by Science.*