Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses

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Abstract

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. © 2008 by Polish Neuroscience Society - PTBUN, Nencki Institute of Experimental Biology.

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Pierret, C., Morrison, J. A., & Kirk, M. D. (2008). Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses. Acta Neurobiologiae Experimentalis. https://doi.org/10.55782/ane-2008-1709

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