Neuronal ceroid lipofuscinoses therapeutic strategies: past, present and future.
Historically, many different therapies have been assessed for their ability to alter disease progression of the Neuronal Ceroid Lipofuscinoses (NCLs). While some treatments have lead to minor improvements, none have been able to arrest disease progression or improve the quality or duration of life. Presently, many new therapeutic strategies, such as chaperone therapy, enzyme replacement therapy, gene therapy, and stem cell therapy, are being investigated for their ability to alter the disease course of the NCLs. This review summarizes previous studied therapies, discusses those currently being evaluated and examines possibilities for future therapies for the treatment of patients with NCL.
Hobert
Judith A
JA
Committee on Molecular Metabolism and Nutrition, Biological Sciences Division, University of Chicago, Chicago, IL 60637, USA.
Dawson
Glyn
G
D000818:Animals; D002986:Clinical Trials as Topic; D019587:Dietary Supplements; D015316:Genetic Therapy; D006801:Humans; D008247:Lysosomes / Q000201:enzymology; D051379:Mice; D018832:Molecular Chaperones / Q000627:therapeutic use; D009472:Neuronal Ceroid-Lipofuscinoses / Q000201:enzymology / Q000235:genetics / Q000628:therapy*; D033581:Stem Cell Transplantation
D018832:Molecular Chaperones