Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

Gene therapy is a powerful tool to treat various central nervous system (CNS) diseases
ranging from monogenetic diseases to neurodegenerative disorders. Adeno-associated …

Abstract Mucopolysaccharidosis type III (MPS III) or Sanfilippo disease is an orphan
inherited lysosomal storage disease and one of the most common MPS subtypes. The …

Mucopolysaccharide diseases are a group of paediatric inherited lysosomal storage
diseases that are caused by enzyme deficiencies, leading to a build-up of …

Delivery of adeno-associated viral vectors (AAVs) to cerebrospinal fluid (CSF) has emerged
as a promising approach to achieve widespread transduction of the central nervous system …

For most lysosomal storage diseases (LSDs), there is no cure. Gene therapy is an attractive
tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which …

Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme
sulfamidase (SGSH), which is responsible for the degradation of heparan sulfate (HS). Build …

Sulfatases are enzymes that catalyze the removal of sulfate from biological substances, an
essential process for the homeostasis of the body. They are commonly activated by the …

Abstract Mucopolysaccharidosis III (Sanfilippo syndrome, MPS III) is caused by lysosomal
enzyme deficiency, which is a rare autosomal recessive hereditary disease. For now, there …