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

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.