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首页> 外文期刊>Current opinion in lipidology >Function of the Niemann-Pick type C proteins and their bypass by cyclodextrin.
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Function of the Niemann-Pick type C proteins and their bypass by cyclodextrin.

机译：Niemann-Pick C型蛋白的功能及其通过环糊精的旁路。

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PURPOSE OF REVIEW: This review summarizes the recent findings on the mechanism of action of the Niemann-Pick type C (NPC) proteins and their bypass by cyclodextrin. RECENT FINDINGS: NPC disease is caused by dysfunction in either the NPC1 or NPC2 protein. These proteins function in the same pathway for the removal of unesterified cholesterol from late endosomes/lysosomes. In NPC-deficient cells, cholesterol derived from the endocytosis of LDLs becomes sequestered in the late endosomes/lysosomes. Recent studies have indicated that these two cholesterol-binding proteins act in tandem in mediating the egress of cholesterol from the late endosomes/lysosomes. Patches of amino acids on NPC1 and NPC2 appear to interact so that the hydrophobic transfer of cholesterol from NPC2 to NPC1 is achieved. Although no effective treatment for NPC disease is currently available, exciting new studies have shown that treatment of NPC-deficient mice with the cholesterol-binding compound, cyclodextrin, reduces the neurodegeneration and markedly extends the life span of Npc1-/- mice, suggesting a potential therapeutic approach for the treatment of individuals with NPC disease. SUMMARY: Experimental data are consistent with a model for the sequential action of the NPC1 and NPC2 proteins in moving cholesterol out of the late endosomes/lysosomes. Recent data demonstrate that treatment of NPC-deficient mice with cyclodextrin extends their life span, thereby suggesting a potential therapy for NPC patients.
机译：综述的目的：这篇综述总结了尼曼-匹克C型（NPC）蛋白的作用机理及其被环糊精绕过的最新发现。最近的发现：NPC疾病是由NPC1或NPC2蛋白的功能障碍引起的。这些蛋白质以相同的途径发挥作用，从晚期内体/溶酶体中去除未酯化的胆固醇。在缺乏NPC的细胞中，源自LDL内吞作用的胆固醇被隔离在晚期的内体/溶酶体中。最近的研究表明，这两种胆固醇结合蛋白协同作用介导晚期内体/溶酶体中胆固醇的释放。 NPC1和NPC2上的氨基酸斑块似乎相互作用，因此实现了胆固醇从NPC2到NPC1的疏水转移。尽管目前尚无有效的NPC疾病治疗方法，但令人兴奋的新研究表明，用胆固醇结合化合物环糊精治疗NPC缺陷小鼠可减少神经变性并显着延长Npc1-/-小鼠的寿命。用于治疗NPC疾病的潜在治疗方法。摘要：实验数据与NPC1和NPC2蛋白在将胆固醇从晚期内体/溶酶体中移出时的顺序作用模型相一致。最近的数据表明，用环糊精治疗NPC缺陷型小鼠可延长其寿命，从而为NPC患者提供了一种潜在的治疗方法。

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