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Peer-reviewedCC BY 4.0International Journal of Molecular Sciences

Targeted Regulation and Treatment Application of Liquid–Liquid Phase Separation in Human Diseases

  1. 1College of Life Science, Northeast Forestry University, Harbin 150040, China
  2. 2School of Future Technology, Northeast Forestry University, Harbin 150040, China
  3. 3Aulin College, Northeast Forestry University, Harbin 150040, China

Abstract

Liquid–liquid phase separation (LLPS) is widely recognized as a fundamental mechanism underlying the formation of membraneless organelles, which are critical for maintaining spatiotemporal precision in cellular function. Dysregulation of LLPS has become increasingly recognized as a key driver underlying the development of multiple human diseases, thereby establishing this process as a rapidly growing focus in biomedical research. In this review, we examine the contributions of LLPS to cellular pathophysiology. We start by outlining the fundamental biophysical and molecular principles that govern LLPS formation. We further explore how aberrant phase transitions contribute to disease mechanisms across different organ systems. Building on this mechanistic framework, we assess current and potential therapeutic strategies aimed at targeting LLPS for clinical intervention. Overall, this review aims to synthesize current understanding of LLPS in disease etiology and to highlight its potential as a novel target for future therapeutic development.

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BibTeX
@article{zhang2026targeted,
  title = {{Targeted Regulation and Treatment Application of Liquid–Liquid Phase Separation in Human Diseases}},
  author = {Yuanxin Zhang and Qingqing Xu and Yujie Liu and Kejia Yao and Nuo Chen and Guangchao Sui and Dangdang Li and Jian Zheng},
  journal = {International Journal of Molecular Sciences},
  year = {2026},
  month = oct,
  doi = {10.3390/ijms27219662},
  url = {https://www.mdpi.com/1422-0067/27/21/9662},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Targeted Regulation and Treatment Application of Liquid–Liquid Phase Separation in Human Diseases
AU  - Yuanxin Zhang
AU  - Qingqing Xu
AU  - Yujie Liu
AU  - Kejia Yao
AU  - Nuo Chen
AU  - Guangchao Sui
AU  - Dangdang Li
AU  - Jian Zheng
PY  - 2026
DA  - 2026/10/28
JO  - International Journal of Molecular Sciences
DO  - 10.3390/ijms27219662
UR  - https://www.mdpi.com/1422-0067/27/21/9662
AB  - Liquid–liquid phase separation (LLPS) is widely recognized as a fundamental mechanism underlying the formation of membraneless organelles, which are critical for maintaining spatiotemporal precision in cellular function. Dysregulation of LLPS has become increasingly recognized as a key driver underlying the development of multiple human diseases, thereby establishing this process as a rapidly growing focus in biomedical research. In this review, we examine the contributions of LLPS to cellular pathophysiology. We start by outlining the fundamental biophysical and molecular principles that govern LLPS formation. We further explore how aberrant phase transitions contribute to disease mechanisms across different organ systems. Building on this mechanistic framework, we assess current and potential therapeutic strategies aimed at targeting LLPS for clinical intervention. Overall, this review aims to synthesize current understanding of LLPS in disease etiology and to highlight its potential as a novel target for future therapeutic development.
SN  - 1422-0067
ER  -