Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

Late infantile neuronal ceroid lipofuscinosis is a progressive childhood ne urodegenerative disorder characterized by intracellular accumulation of aut ofluorescent material resembling lipofuscin in neuronal cells. This report summarizes the new therapies under consideration for late infantile neurona l ceroid lipofuscinosis, with a focus on strategies for in vivo gene therap y for the retinal and central nervous system manifestations of the disease.