Journal
ARCHIVES OF NEUROLOGY
Volume 58, Issue 11, Pages 1793-1798Publisher
AMER MEDICAL ASSOC
DOI: 10.1001/archneur.58.11.1793
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Late infantile neuronal ceroid lipofuscinosis is a progressive childhood neurodegenerative disorder characterized by intracellular accumulation of autofluorescent material resembling lipofuscin in neuronal cells. This report summarizes the new therapies under consideration for late infantile neuronal ceroid lipofuscinosis, with a focus on strategies for in vivo gene therapy for the retinal and central nervous system manifestations of the disease.
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