Huntington’s Disease Successfully Treated
Doctors have successfully treated Huntington’s disease for the first time using an experimental gene therapy, slowing its progression by 75% in a trial of 29 patients. The treatment, delivered thought 12-18 hours of delicate brain surgery, uses a modified virus to insert DNA that reduces production of the toxic huntingtin protein, protecting brain cells. Patients showed remarkable improvements, such as continuing to walk when expected to need a wheelchair and even returning to work. Experts hailed the results as ‘spectacular’ and potentially life-changing, offering decades of good quality life and the possibility of preventing symptoms altogether if used earlier. While expensive and complex, researchers believe this breakthrough marks the start of transformative therapies for Huntington’s and related genetic diseases.
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