Mucopolysaccharidosis type IVA (MPS IVA, or Morquio syndrome type A) is an inherited
metabolic lysosomal disease caused by the deficiency of the N-acetylglucosamine-6-sulfate …

Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders
with complex clinical phenotypes that include both systemic and central nervous system …

Impaired degradation of glycosaminoglycans (GAGs) with consequent intralysosomal
accumulation of undegraded products causes a group of lysosomal storage disorders …

The majority of lysosomal storage diseases affect the brain. Treatment of the brain with
intravenous enzyme replacement therapy is not successful, because the recombinant …

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 …

Rare monogenic disorders such as lysosomal diseases have been at the forefront in the
development of novel treatments where therapeutic options are either limited or unavailable …

Sanfilippo syndrome type B (mucopolysaccharidosis III B, MPS III B) is an autosomal
recessive, neurodegenerative disease of children, characterized by profound mental …

Mucopolysaccharidosis type IIIB (Sanfilippo syndrome) is a lysosomal storage disease
caused by a genetic defect in the production of α-N-acetylglucosaminidase. This results in …

Abstract Mucopolysaccharidosis IIIB (MPS IIIB; Sanfilippo syndrome type B) is characterized
by profound neurological deterioration. Because a murine model of MPS IIIB disease is …

We evaluated the ability of an integrase (IN)-defective self-inactivating lentiviral vector
(sinLV) for the delivery of human immunodeficiency virus-1 (HIV-1) envelope sequences in …