Strategies to treat neurodegeneration in neuronal ceroid lipofuscinosis: a view onto the retina.

Neural regeneration research(2023)

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摘要
Neuronal ceroid lipofuscinosis(NCL),also known as Batten disease,is the umbrella term for a group of neurodegenerative lysosomal storage disorders with onset mainly in childhood.The total 13 genetically distinct NCLs are caused by mutations in genes encoding soluble or transmembrane proteins,and have been classified according to the affected gene into CLN1 to CLN8 and CLN10 to CLN14.
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关键词
neuronal ceroid lipofuscinosis,neurodegeneration
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