化学
细胞内
肽
体内
脂肪酸
细胞通透性
生物化学
生物
生物技术
作者
Chuanliang Zhang,Guozhen Dong,Xiao Qing Wu,Jin Chen,Yanqing Wang,Liyan Gong,Xianmin Yang,Yiying Shi,Zongwen Gu,Xiang Gao,Yaning Zheng,Han Wu,Ke Zheng,Xiaochun Liu,Yuchao Gu
标识
DOI:10.1021/acs.jmedchem.5c00147
摘要
Fatty acid derivatization is a promising strategy for discovering long-acting peptide therapeutics, but intracellular targeting remains challenging due to insufficient membrane permeability. Here, we report a dual fatty acid conjugation approach to develop ultralong-acting inhibitors of intracellular PTPN1/2. By conjugating a long-chain fatty acid/diacid to the N-terminus and a medium-chain fatty acid/diacid to Lys 2 of BimBH3 peptide, we achieved efficient cell permeability and uptake for intracellular target inhibition and metabolic stability for long-acting in vivo efficacy. The optimized analogue D6 exhibited potent dual PTPN1/2 inhibition (IC 50 = 107.6 nM and 3375 nM), 40-fold improved DPP-IV stability, and prolonged plasma half-life (>200 h) in rats following sc administration. D6 exhibited efficient cell permeability, restored insulin signaling in HepG2 cells and demonstrated once-weekly glycemic control in db/db mice. Molecular docking revealed key interactions with PTPN1/2 active sites. This work demonstrates a strategy for designing cell-permeable, long-acting peptide inhibitors of intracellular targets.
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