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

Systemic injection of an adenovirus vector into adult mice resulted in pathological
improvements in multiple visceral organs of mice with mucopolysaccharidosis VII; however …

Mucopolysaccharidosis I (MPS I) and MPS VII are due to deficient activity of the
glycosaminoglycan-degrading lysosomal enzymes α-l-iduronidase and β-glucuronidase …

Mucopolysaccharidosis (MPS) VII is a lysosomal storage disease due to deficient activity of
β-glucuronidase (GUSB), and results in glycosaminoglycan accumulation. Skeletal …

For many metabolic diseases, early correction of the inherited deficiency is required to
prevent long-term sequelae. We examined the ability of adeno-associated virus (AAV) to …

Mucopolysaccharidosis VII (MPS VII) is a lysosomal storage disease caused by deficient β-
glucuronidase (GUSB) activity. Accumulation of glycosaminoglycans (GAGs) in bone …

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 …

Recombinant adenoviruses expressing human b-glucuronidase (AxCAhGUS) and CTLA-4Ig
(AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine …

For many inborn errors of metabolism, early treatment is critical to prevent long-term
developmental sequelae. We have previously shown that systemic treatment of neonatal …

Mucopolysaccharidosis type IIIA (MPS IIIA) is a heritable glycosaminoglycan (GAG) storage
disorder which is characterised by lysosomal accumulation of heparan sulphate, secondary …