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5 February 2026

Targeting the UPR with Small Molecules: Emerging Strategies for Immune Regulation

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and
1
Brigham Regenerative Medicine Center, Brigham and Women’s Hospital, Boston, MA 02215, USA
2
Division of Cardiovascular Medicine, Heart and Vascular Institute, Department of Medicine, Harvard Medical School, Boston, MA 02215, USA
3
Harvard Stem Cell Institute, Cambridge, MA 02138, USA
4
Department of Pathology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA 02215, USA
This article belongs to the Special Issue Small-Molecule Targeted Drugs

Abstract

The unfolded protein response (UPR) is a highly conserved adaptive mechanism that restores endoplasmic reticulum (ER) homeostasis under stress. Beyond its canonical roles in proteostasis, the UPR has emerged as a central regulator of immune responses across diverse contexts, including infection, inflammation, cancer, and autoimmunity. IRE1α, PERK, and ATF6 are three principal UPR sensors that coordinate complex signaling networks to regulate antigen presentation, cytokine production, and immune cell differentiation. This review highlights the molecular mechanisms by which small molecules target the UPR to modulate immune responses. In addition, we highlight stress granules (SGs) and the prevalence of protein–protein interactions mediated by intrinsically low-complexity domains (LCDs) in the UPR as potential new avenues for immune modulation. Finally, we discuss future directions for leveraging UPR modulation in immunotherapy, infectious disease, and chronic inflammatory disorders.
Keywords:
ER; UPR; small molecules; immunity; SG; LCD

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