Lysosomal storage disorders and their impact upon the central nervous system:Lysosomal storage disorders(LSDs)are a group of over 70 rare inherited metabolic disorders(Platt et al.,2018).They are caused by dysfunction...Lysosomal storage disorders and their impact upon the central nervous system:Lysosomal storage disorders(LSDs)are a group of over 70 rare inherited metabolic disorders(Platt et al.,2018).They are caused by dysfunction of lysosomes,organelles that contain enzymes responsible for digesting macromolecules.In functional lysosomes,these enzymes break down complex substrates,and the resulting fragments are recycled.Individual LSDs are caused by mutations in genes that encode lysosomal enzymes or other proteins crucial for lysosome function(Platt et al.,2018).展开更多
文摘Lysosomal storage disorders and their impact upon the central nervous system:Lysosomal storage disorders(LSDs)are a group of over 70 rare inherited metabolic disorders(Platt et al.,2018).They are caused by dysfunction of lysosomes,organelles that contain enzymes responsible for digesting macromolecules.In functional lysosomes,these enzymes break down complex substrates,and the resulting fragments are recycled.Individual LSDs are caused by mutations in genes that encode lysosomal enzymes or other proteins crucial for lysosome function(Platt et al.,2018).