Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.

Abstract

Over the past three decades, enzyme therapy for lysosomal storage diseases has moved from an academic pursuit to direct delivery of effective clinical care for affected patients and families. This success is based on understanding the complexities of lysosomal biogenesis, lysosomal hydrolase sorting and hydrolytic requirements, and the target sites of… (More)

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Cite this paper

@article{Grabowski2003EnzymeTF, title={Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.}, author={G. Grabowski and Robert J. Hopkin}, journal={Annual review of genomics and human genetics}, year={2003}, volume={4}, pages={403-36} }