"Promising Results from NIH Clinical Trial of Gene Therapy for Giant Axonal Neuropathy"
An experimental gene therapy for giant axonal neuropathy (GAN) has shown promise in a clinical trial led by the National Institutes of Health (NIH), with results published in the New England Journal of Medicine. The therapy, which uses a modified virus to deliver functional copies of the defective GAN gene to nerve cells, was well tolerated and appeared to slow the rate of motor function decline in some patients. Additionally, some patients showed signs of regeneration of sensory nerves. The study marks the first time that a sensory nerve affected in a genetic degenerative disease has been shown to be rescued with gene therapy. The next phase of the trial will focus on evaluating the therapy's safety and efficacy in younger children or those in an earlier stage of the disease.