Sanfilippo syndrome type B (MPS IIIB) is a lysosomal storage disease resulting from a
deficiency of N-acetyl-glucosaminidase (NAGLU) activity. In an attempt to correct the …

Abstract Mucopolysaccharidosis type IIIB (MPS IIIB) or Sanfilippo Syndrome type B is a
lysosomal storage disease resulting from the deficiency of N-acetyl glucosaminidase …

Sanfilippo syndrome is a mucopolysaccharidosis (MPS) caused by a lysosomal enzyme
defect interrupting the degradation pathway of heparan sulfates. Affected children develop …

Gene therapy is a promising therapeutic alternative for Lysosomal Storage Disorders (LSD),
as it is not necessary to correct the genetic defect in all cells of an organ to achieve …

Sanfilippo syndrome type B (mucopolysaccharidosis IIIB, MPS IIIB) is a lysosomal storage
disease resulting from deficiency of N-acetyl-glucosaminidase (NAGLU) activity. To …

Mucopolysaccharidosis type IIIB syndrome (Sanfilippo disease) is a rare autosomic recessif
disorder caused by mutations in the α-N-acetylglucosaminidase (NAGLU) gene coding for a …

Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB [MPS IIIB]) is a lysosomal
storage disorder primarily affecting the brain that is caused by a deficiency in the enzyme α …

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 …

Mucopolysaccharidosis IIIB (MPS IIIB) is a lysosomal storage disorder caused by mutations
in NAGLU, the gene encoding α-N-acetylglucosaminidase. The disease is characterized by …

Background Finding efficient central nervous system (CNS) delivery approaches has been
the major challenge facing therapeutic development for treating diseases with global …