Diseases
Groundbreaking Gene Editing Trial for Rare Disease GSD1a Begins at UConn Health and Connecticut Children's
Jennifer Walker UConn Health and Connecticut Children's have made history by dosing the first patient in a clinical trial using gene editing to treat glycogen storage disease type 1a (GSD1a). This global first marks the third "first-in-human" trial for GSD1a conducted by this research team. The