Kris Saha, PhD, an Associate Professor at the University of Wisconsin-Madison USA, discusses the recent work related to correcting GAA mutations. These mutations are present in patients with Pompe disease. Gene therapy, including technology such as CRISPR, ca be efficacious for managing Pompe disease. Dr Saha notes that patients who are excluded from gene addition therapeutic trials, such as CRIM-negative patients, should participate in genome editing clinical trials.