Gene Therapy in Patients With MPS IIIA

Gene Therapy in Patients With MPS IIIA by Madaline Spencer| Published on: Mar 18, 2026 Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses hematopoietic stem cell gene therapy (HSCGT) in patients with mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo syndrome type A). https://checkrare.com/gene-therapy-in-patients-with-mps-iiia/