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Shift of paradigm on HPBCD as cholesterol sequestering agent in cells

机译：HPBCD上的范式转移为细胞中胆固醇隔离剂

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It is well known for the readers of the Cyclodextrin News [1-4] that BCDs, especially methyl BCD and the less toxic HPBCD remove cholesterol accumulated in cells of patients 1. with Niemann Pick type C1 disease, where the lack of npc1 and/or npc2 genes responsible for the synthesis of NPC1 and NPC2 proteins transporting cholesterol out of late endosomes cause the lysosomal storage disorder of cholesterol resulting in symptoms of childhood neurodegeneration and these symptoms are improved by HPBCD treatment; 2. with cancer, such as leukemia, where HPBCD treatment disturbs cholesterol homeostasis; 3. with atherosclerosis, where the cholesterol efflux from atherosclerotic plaques is increased via mobilization of cholesterol with HPBCD; 4. with cystic fibrosis, where the malfunction of cholesterol-enriched pulmonary surfactant can be restored by methyl BCD.
机译：Cyclodextrin News的读者[1-4]众所周知，BCD，尤其是甲基BCD和毒性较小的HPBCD消除了在患者的细胞中累积的胆固醇1.患有NIEMANN PICK型C1疾病，缺乏NPC1和// 或NPC2基因负责合成NPC1和NPC2蛋白从后期内体内输送胆固醇的基因会导致胆固醇的溶酶体储存障碍导致儿童神经变性的症状通过HPBCD治疗改善这些症状; 2.癌症，例如白血病，HPBCD治疗干扰了胆固醇稳态; 3.随着动脉粥样硬化，通过动员HPBCD，通过动员胆固醇来增加动脉粥样硬化斑块的胆固醇外排。 4.伴有囊性纤维化，可以通过甲基BCD恢复富含胆固醇的肺表面活性剂的故障。

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