DL001 是一种依赖于 FKBP12 的雷帕霉素类似物，选择性的 mTORC1 抑制剂，IC50 为 74.9 pM。 在细胞系中，DL001 有效抑制升高的 mTORC1 活性并恢复正常基因表达至缺乏功能性结节性硬化症复合体的细胞。在 C57BL/6J 小鼠体内，DL001 抑制 mTORC1 信号传导，但不影响葡萄糖体内平衡，并且显着降低脂质代谢和体内免疫系统而没有其他副作用。
DL001, a FKBP12-dependent rapamycin analog, is a selective mechanistic Target Of Rapamycin Complex 1 (mTORC1) inhibitor with an IC50 of 74.9 pM. In cells, DL001 efficiently represses elevated mTORC1 activity and restores normal gene expression to cells lacking a functional tuberous sclerosis complex. DL001 inhibits mTORC1 signaling without impairing glucose homeostasis and with substantially reduced and no side effects on lipid metabolism and the immune system in vivo in C57BL/6J mice[1]. mTORC1|74.9 pM (IC50)
[1]. Schreiber KH , et al. A novel rapamycin analog is highly selective for mTORC1 in vivo. Nat Commun. 2019 Jul 19;10(1):3194.
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