Dear Editor,Gaucher disease(GD)is the most common lysosomal storage disease(LSD)caused by an insufficiency of the lysosomal enzyme glucocerebrosidase(GCase)[1].GCase insufficiency produces the excessive lysosomal accu...Dear Editor,Gaucher disease(GD)is the most common lysosomal storage disease(LSD)caused by an insufficiency of the lysosomal enzyme glucocerebrosidase(GCase)[1].GCase insufficiency produces the excessive lysosomal accumulation of unmetabolized glycolipid substrates including glucosylceramide(GlcCer),leading to the disruption of the structure and function of tissues and organs,includingthe blood system,viscera,brain,boncs,and cartilage.展开更多
基金the National Natural Science Foundation of China(81771416,81650110527,and 8197100)Shanghai Municipal Commission of Health and Family Planning(201740153)Key Discipline of Chongming District,Shanghai,China,2018.
文摘Dear Editor,Gaucher disease(GD)is the most common lysosomal storage disease(LSD)caused by an insufficiency of the lysosomal enzyme glucocerebrosidase(GCase)[1].GCase insufficiency produces the excessive lysosomal accumulation of unmetabolized glycolipid substrates including glucosylceramide(GlcCer),leading to the disruption of the structure and function of tissues and organs,includingthe blood system,viscera,brain,boncs,and cartilage.
