体外将葡萄糖脑苷脂酶导入戈谢病单核细胞。
Incorporation of glucocerebrosidase into Gaucher's disease monocytes in vitro.
作者信息
Dale G L, Kuhl W, Beutler E
出版信息
Proc Natl Acad Sci U S A. 1979 Jan;76(1):473-5. doi: 10.1073/pnas.76.1.473.
Abstract
Several carriers were evaluated for use in the delivery of exogenous glucocerebrosidase to monocytes from Gaucher's disease patients. Only gamma globulin-coated, resealed erythrocytes proved to be an effective vehicle for enzyme delivery. Glucocerebrosidase added in this manner normalized intracellular enzyme levels for at least 18 hr. In this model system for the study of enzyme replacement therapy, soluble enzyme, enzyme in uncoated resealed erythrocytes, and enzyme incorporated into liposomes were ineffective.
摘要
对几种载体进行了评估，以用于将外源性葡萄糖脑苷脂酶递送至戈谢病患者的单核细胞。只有γ球蛋白包被的、重新封闭的红细胞被证明是一种有效的酶递送载体。以这种方式添加的葡萄糖脑苷脂酶使细胞内酶水平至少在18小时内恢复正常。在这个用于研究酶替代疗法的模型系统中，可溶性酶、未包被的重新封闭红细胞中的酶以及掺入脂质体中的酶均无效。
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