Fatal neurodegenerative disease in kids also affects the bowel—gene therapy may help

Researchers at Washington University School of Medicine in St. Louis have described the neurodegeneration that occurs in the nervous system of the bowel in Batten disease, a rare and fatal genetic condition. In their latest study, a team led by Jonathan Cooper, Ph.D., professor of pediatrics at WashU Medicine, has shown that gene therapy to the bowel in mice modeling Batten disease reduced symptoms and extended lifespan.