Completed Cells, Biochemistry & Physiology Genetics & Molecular Biology

Exploring accessible nodes in the Unfolded Protein Response

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AI plain-English summary

Every cell in the body has a quality-control system that flags and fixes misshapen proteins, and this project will map four little-understood parts of that machinery. As we age, this system falters, allowing toxic protein clumps to accumulate—a hallmark of diseases like Alzheimer’s, Parkinson’s, and motor neurone disease. The research targets two specific control points: the molecular switch that slows down protein production during stress (eIF2α), and the mechanism that inactivates a key chaperone protein called BiP. By working out the precise shapes and chemical interactions of these components, the team aims to identify weak points where a drug could nudge the system back toward healthy function. This is fundamental science—no immediate treatment will come from it. But similar work on protein-folding pathways has already spawned experimental drugs for rare genetic disorders. A clearer blueprint of these stress-response nodes could eventually let researchers design molecules that prevent the cellular pile-up of misfolded proteins, potentially slowing the progression of age-related brain diseases.

View original technical description
Protein folding homeostasis (proteostasis) in the endoplasmic reticulum (ER) intercedes in biological processes with consequences to diseases of aging. Cells cope with ER stress by addressing features common to most unfolded (and misfolded) proteins. Implementing the apparatus for this Unfolded Protein Response (UPR) entails tradeoffs that affect fitness in circumstance-dependent ways. Our research program is predicated on the notion that a detailed understanding of the UPR will identify failures of homeostasis that may be exploited therapeutically. We shall focus on four promising and underexplored nodes. The first two emerge from study of the signaling pathway by which cells downregulate global protein synthesis in response to ER stress, which hinges on phosphorylated translation initiation factor, eIF2a. We seek a detailed biochemical and structural understanding of eIF2αP dephosphorylation by PPP1R15-containing holophosphatases (whose inhibition promotes resistance to ER stress) and the action of eIF2B, a guanine nucleotide exchange factor [the target of eIF2(αP)]. Nodes 3 and 4 concern the machinery that regulates proteostasis by inactivating the ER chaperone BiP through enforced oligomerization or covalent modification. Delineating the UPR’s fundamentals is the foundation for the rapidly-advancing research into the experimental pathology of ER stress and fuels this important translational effort.

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Researchers

David Ron (EPMC Awardee)

Related Research

Grants with similar aims, by meaning.

Protein folding homeostasis - from mechanisms to interventions
The physiology and pathophysiology of unfolded protein responses.
Structural and mechanistic understanding of Unfolded Protein Response (UPR) regulation by ER Hsp70 resident chaperone BIP.
Structural and mechanistic studies of signal activation, transduction across the ER membrane, and propagation of the unfolded protein response and its link with other signalling pathways
ER stress signalling by the UPR activator IRE1 and its regulation by BiP

Original classification

Principal Research Fellowship Renewal

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