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 …

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 …

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 …

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

Murine models of lysosomal storage diseases provide an opportunity to evaluate the
potential for gene therapy to prevent systemic manifestations of the disease. To determine …

Mucopolysaccharidosis type I (MPS I), a deficiency in the lysosomal enzyme alpha-L-
iduronidase (IDUA), is characterized by skeletal abnormalities, hepatosplenomegaly and …

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

Most lysosomal storage diseases, including mucopolysaccharidosis, affect the central
nervous system (CNS). They often induce severe and progressive mental retardation …

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

Mucopolysaccharidosis type VII (Sly syndrome) is a lysosomal storage disease caused by
inherited deficiency of the lysosomal enzyme β-glucuronidase. A murine model of this …