The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade
EM Xing, VW Knox, PA O'Donnell, T Sikura… - Molecular genetics and …, 2013 - Elsevier
Mucopolysaccharidosis (MPS) VII is a lysosomal storage disease due to deficient activity of
β-glucuronidase (GUSB), and results in glycosaminoglycan accumulation. Skeletal
manifestations include bone dysplasia, degenerative joint disease, and growth retardation.
One gene therapy approach for MPS VII involves neonatal intravenous injection of a gamma
retroviral vector expressing GUSB, which results in stable expression in liver and secretion
of enzyme into blood at levels predicted to be similar or higher to enzyme replacement …
β-glucuronidase (GUSB), and results in glycosaminoglycan accumulation. Skeletal
manifestations include bone dysplasia, degenerative joint disease, and growth retardation.
One gene therapy approach for MPS VII involves neonatal intravenous injection of a gamma
retroviral vector expressing GUSB, which results in stable expression in liver and secretion
of enzyme into blood at levels predicted to be similar or higher to enzyme replacement …