Enzyme replacement therapy in α-mannosidosis guinea-pigs

Abstract

α-Mannosidosis is a lysosomal storage disorder caused by deficient activity of lysosomal α-mannosidase and is characterised by massive accumulation of mannose-containing oligosaccharides in affected individuals. Patients develop behaviour and learning difficulties, skeletal abnormalities, immune deficiency and hearing impairment. Disease in α-mannosidosis guinea-pigs resembles the clinical, histopathological, biochemical and molecular features of the human disease. We have used the guinea-pig model to investigate efficacy of enzyme replacement therapy as a treatment for α-mannosidosis. Intravenous recombinant human lysosomal α-mannosidase, administered at a dose of 1 mg/kg, was cleared from ..