Murray G J, Doebber T W, Shen T Y, Wu M S, Ponpipom M M, Bugianesi R L, Brady R O, Barranger J A
Biochem Med. 1985 Oct;34(2):241-6. doi: 10.1016/0006-2944(85)90117-6.
Human placental beta-glucocerebrosidase modified by covalent attachment of N2-(N2, N6-bis [3-(alpha-D-mannopyranosylthio)propionyl]-L- lysyl)-N6-[3-(alpha-D-mannopyranosylthio)propionyl]-L-lysine was administered to rats by intravenous injection. Comparison of enzyme distribution in isolated liver cell populations indicates an increase in enzyme-specific activity of 18-fold in nonparenchymal cells and only 1.5-fold to hepatocytes compared to uninjected control animals. This macrophage-specific delivery of an active lysosomal enzyme has potential for application in enzyme replacement trials.
通过静脉注射将经共价连接N2-(N2,N6-双[3-(α-D-甘露吡喃糖基硫代)丙酰基]-L-赖氨酸)-N6-[3-(α-D-甘露吡喃糖基硫代)丙酰基]-L-赖氨酸修饰的人胎盘β-葡萄糖脑苷脂酶给予大鼠。对分离的肝细胞群体中酶分布的比较表明，与未注射的对照动物相比，非实质细胞中酶的比活性增加了18倍，而肝细胞中仅增加了1.5倍。这种活性溶酶体酶的巨噬细胞特异性递送在酶替代试验中具有应用潜力。
