Overcoming the blood-brain barrier: therapy delivery for Sanfilippo

Disease correction by combined neonatal intracranial AAV and

Toward the successful delivery of lysosomal enzymes across the

Mannose 6-Phosphate Receptor–mediated Transport of Sulfamidase

Emerging therapies for neurodegenerative lysosomal storage disorders ‐ from concept to reality - Hemsley - 2011 - Journal of Inherited Metabolic Disease - Wiley Online Library

Gene Therapy Has Potential to Treat Underlying Cause of Sanfilippo

Frontiers Delivering gene therapy for mucopolysaccharide diseases

Full article: Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome)

Blood-Brain Barrier Opening - Focused Ultrasound Foundation

Evolving new-age strategies to transport therapeutics across the