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• W2004999768 abstract "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." @default.
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• W2004999768 date "2001-11-01" @default.
• W2004999768 modified "2023-09-27" @default.
• W2004999768 title "Feasibility of Gene Therapy for Late Neuronal Ceroid Lipofuscinosis" @default.
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• W2004999768 doi "https://doi.org/10.1001/archneur.58.11.1793" @default.
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