Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses
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Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments are considered across the LSDs and extended to therapies for the NCLs. It is likely that a combination of approaches will produce the most beneficial clinical outcome for treatment of pathologies displayed by the NCLs.
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Mark D. Kirk, Division of Biological Sciences, University of Missouri, Columbia, MO 65211, USA