Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of the galactosamine
(N-acetyl)-6-sulfatase (GALNS) enzyme responsible for the degradation of specific …

Mucopolysaccharidosis type IVA (MPS IVA) is due to the deficiency of GALNS (N-
acetylgalactosamine 6-sulfate sulfatase) and is characterized by systemic skeletal dysplasia …

Previous treatment of mucopolysaccharidosis type VII mice (Sly syndrome) with AAV vectors
has resulted in increased levels of β-glucuronidase (GUS) enzyme in some tissues with …

Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage
disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency …

Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is caused by a deficiency of the
N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) enzyme, leading to the accumulation of …

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 …

Adeno-associated virus (AAV) vector-based therapies can effectively correct some disease
pathology in murine models with mucopolysaccharidoses. However, immunogenicity can …

Mucopolysaccharidosis (MPS) IIIB is characterized by mild somatic features and severe
neurological diseases leading to premature death. No definite treatment is available for MPS …

Neonatal gene transfer using adenovirus vectors expressing human β-glucuronidase
(AxCAhGUS) resulted in pathological improvement in multiple visceral organs of mice with …

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