Cell-membrane proteins are critical mediators of signal transduction, playing essential roles in disease occurrence and progression. The emerging LYTACs (Lysosome-targeting chimeras) technology combines drug-targeting strategies with lysosomal degradation, providing a novel approach to drug development and offering new possibilities for disease therapy. However, the clinical applicability of current LYTAC degraders is limited by the variable expression of lysosome-targeting receptors (LTRs) in tissues. To overcome this limitation, we herein hijacked a YXXÃ sorting signal that derived from lysosome-associated membrane protein 2a (LAMP-2a) to develop a signal aptamer platform (SApt), which exhibits high specificity for targeting membrane proteins and inducing efficient lysosomal degradation. SApts were synthesized by conjugating the YXXÃ signal peptide to the aptamer's terminus through a click reaction. Our study demonstrated that SApts efficiently degrade disease-associated membrane proteins, such as PTK7, Met, and NCL, based on the inherent signals rather than specific LTR. The potent antitumor efficacy of SApts was further confirmed in a xenograft tumor model, where in vivo degradation of PTK7 was observed. Collectively, our work provides insights into the development of a simple and universal lysosomal degradation platform with potential translational value in clinical treatment.
Click-constructed modular signal aptamer chimeras enable receptor-independent degradation of membrane proteins.
利用点击反应构建的模块化信号适体嵌合体能够实现膜蛋白的受体非依赖性降解
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作者:Xie Wanlin, Sun Weidi, Li Qin, Dang Yang, Ma Lele, Liu Yuan, Zhang Hui, Qu Fengli, Tan Weihong
| 期刊: | Proceedings of the National Academy of Sciences of the United States of America | 影响因子: | 9.100 |
| 时间: | 2025 | 起止号: | 2025 May 27; 122(21):e2424500122 |
| doi: | 10.1073/pnas.2424500122 | 研究方向: | 信号转导 |
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