药品
癌症
靶向给药
癌症研究
药物输送
化学
翻译(生物学)
肽
蛋白质降解
纳米纤维
水解降解
降级(电信)
药理学
癌细胞
生物利用度
有效载荷(计算)
前药
肿瘤细胞
免疫系统
癌症治疗
纳米技术
机制(生物学)
医学
药物发现
药物开发
作者
Yuchen Yang,Chunrong Yang,Zheng Fu,Meichen Pan,Libing Wu,Hongwei Hou,Jinghong Li
标识
DOI:10.1002/adhm.202505105
摘要
ABSTRACT Proteolysis‐targeting chimeras (PROTACs) have emerged as a promising strategy for the selective degradation of oncoproteins, offering significant potential in cancer therapy, yet their clinical translation is impeded by the inherent limitations, including high hydrophobicity, poor cellular permeability, and inadequate tumor‐targeting efficiency. To address these challenges, we developed a rationally engineered peptide conjugate, Nap‐Lys(ARV‐771)‐Tyr(H 2 PO 3 )‐Phe‐Phe‐OH (NapYp‐ARV), to enhance the bioavailability and therapeutic efficacy of PROTACs through sequential enzyme‐instructed self‐assembly (EISA) and activation. Upon intratumoral phosphatase‐mediated dephosphorylation, NapYp‐ARV self‐assembles into nanofibers that enhance tumor accumulation. Following cellular internalization, carboxylesterase‐catalyzed hydrolysis liberates the PROTAC payload ARV‐771, inducing potent BRD4 degradation and consequent apoptosis. In vivo, NapYp‐ARV administration achieved higher tumor drug concentration than free ARV‐771, driving robust protein degradation and tumor regression while maintaining hematological and histological safety. Collectively, this EISA‐based delivery platform offers a versatile strategy to advance targeted PROTAC therapies.
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