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药物靶向内在无序蛋白质和生物分子凝聚物的当前观点。

Current perspectives in drug targeting intrinsically disordered proteins and biomolecular condensates.

作者信息

Qin Caolitao, Wang Yun-Long, Zheng Jian, Wan Xiang-Bo, Fan Xin-Juan

机构信息

Department of Radiation Oncology, The Sixth Affiliated Hospital, Sun Yat-Sen University, Guangzhou, Guangdong, 510655, People's Republic of China.

GuangDong Provincial Key Laboratory of Colorectal and Pelvic Floor Diseases, The Sixth Affiliated Hospital, Sun Yat-Sen University, Guangzhou, Guangdong, 510655, People's Republic of China.

出版信息

BMC Biol. 2025 May 6;23(1):118. doi: 10.1186/s12915-025-02214-x.

Abstract

Intrinsically disordered proteins (IDPs) and biomolecular condensates are critical for cellular processes and physiological functions. Abnormal biomolecular condensates can cause diseases such as cancer and neurodegenerative disorders. IDPs, including intrinsically disordered regions (IDRs), were previously considered undruggable due to their lack of stable binding pockets. However, recent evidence indicates that targeting them can influence cellular processes. This review explores current strategies to target IDPs and biomolecular condensates, potential improvements, and the challenges and opportunities in this evolving field.

摘要

内在无序蛋白质(IDPs)和生物分子凝聚物对细胞过程和生理功能至关重要。异常的生物分子凝聚物可导致癌症和神经退行性疾病等疾病。包括内在无序区域(IDRs)在内的IDPs,由于缺乏稳定的结合口袋,以前被认为是不可成药的。然而,最近的证据表明,针对它们可以影响细胞过程。本综述探讨了针对IDPs和生物分子凝聚物的当前策略、潜在改进以及这一不断发展的领域中的挑战和机遇。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/394b/12054275/1caf71159baf/12915_2025_2214_Fig1_HTML.jpg

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