Enzyme-replacement therapy in lysosomal storage diseases / Julie Bielicki.

Abstract

Describes experiments involving two animal models; the MPS VI cat and the fucosidosis dog to demonstrate the effectiveness of enzyme-replacement therapy (ERT) in lysosomal storage diseases (LSD). Pathology in these animal models closely parallels that observed in the human condition. In terms of therapeutic strategies, the results described in this thesis indicate that the use of same-species enzyme is superior to non-same-species enzyme. ERT used on the fucosidosis dog appeared to delay the onset of neurological symptoms. Although technically difficult to produce, chimeric proteins may provide a means of treatment for neurological symptoms in LSD.