Remarkably, it is less than 50 years since Christian de Duve first described the lysosome and proposed the concept of enzyme replacement therapy (ERT) for lysosomal storage diseases (LSDs). Following his ground-breaking discovery, tremendous advances have been made in our understanding of lysosomal physiology. In particular, we have gained insight into the function and trafficking of the many lysosomal enzymes and associated proteins that are involved in the cellular recycling of complex macromolecules. Despite these advances, and the increased appreciation of the role of lysosomes and lysosomal enzymes in health and disease, it is only recently that ERT has become available. Even now, ERT has been developed for only a small number of the approximately 50 recognized LSDs.
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