Pilot Scale Isolation of Ceramide Trihexosidase from Human Plasma and Clinical Evaluation of Enzyme Replacement Therapy in Fabry's Disease.

摘要

The research concerns the development of a procedure for the purification of ceramide trihexosidase from Cohn Fr IV-1of human plasma,modification of the procedure for pilot-scale isolation of this lysosomal enzyme,development of a method of storage,and clinical evaluation of the enzyme for replacement therapy in Fabry's disease.