Delivery of sulfamidase to the brain
MPS IIIA (Sanfilippo A) is a disease in which a key enzyme is missing in cells resulting in the accumulation of a type of complex sugar called a glycosaminoglycan in … Continued
AAV2/8-mediated expression of modified sulphamidase, liver targeting for improved secretion and brain delivery in IIIA Huntaway dog.
This is a pre-clinical trial of a new gene therapy approach for Sanfilippo syndrome type A. The therapy treats brain disease in mice with Sanfilippo type A (MPS IIIA; Sorrentino … Continued