Browsing Genetics Area Program (MU) by Title "Ex vivo gene therapy for the preservation of retinal and central nervous system structure and function in a canine model of CLN2 neuronal ceroid lipofuscinosis"
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Ex vivo gene therapy for the preservation of retinal and central nervous system structure and function in a canine model of CLN2 neuronal ceroid lipofuscinosis
(University of Missouri--Columbia, 2016)The neuronal ceroid lipofuscinoses (NCLs) are an inherited group of related lysosomal storage disorders. The NCLs are characterized in general by the accumulation of autofluorescent lysosomal storage material (ceroid-type ...