Treatment of lysosomal storage disorders: focus on the neuronal ceroid-lipofuscinoses.
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.
Pierret
Chris
C
Division of Biological Sciences, University of Missouri, Columbia, MO 65211, USA.
Morrison
Jason A
JA
Kirk
Mark D
MD
D000818:Animals; D057487:Enzyme Therapy; D015316:Genetic Therapy / Q000639:trends*; D006801:Humans; D009472:Neuronal Ceroid-Lipofuscinoses / Q000188:drug therapy* / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000639:trends*; D004798:Enzymes
D004798:Enzymes