Mayo researchers reveal gene therapy path for treating children with rare, fatal genetic disease

A gene therapy strategy developed by Mayo Clinic researchers could offer a potential treatment for a rare and fatal genetic disease that often sickens babies in their first days of life. The disease, propionic acidemia, occurs in 1 in 100,000 live births in the U.S. There is no cure. "As soon as the babies start [...]
Source: News from Mayo Clinic - Category: Databases & Libraries Source Type: news