Evidence map›Paper›PMID 41289394›Full record

ArticleProceedings of the National Academy of Sciences of the United States of America2025

Drug repurposing screen identifies an HRI activating compound that promotes adaptive mitochondrial remodeling in MFN2-deficient cells.

Prerona Bora, Mashiat Zaman, Samantha Oviedo, Sergei Kutseikin, Nicole Madrazo, Prakhyat Mathur, Meera Pannikkat, Sophia Krasny, Rama Aldakhlallah, Alan Chu and 4 more

Abstract read
In one paragraph

Article in Proceedings of the National Academy of Sciences of the United States of America, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

14 authors.

Prerona Bora *Department of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0002-3489-6146
Mashiat Zaman *Department of Biochemistry and Molecular Biology, Cummings School of Medicine, University of Calgary, Calgary, AB T2N 4N1, Canada.ORCID 0000-0002-1385-6591
Samantha OviedoDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.
Sergei KutseikinDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.
Nicole MadrazoDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0003-3731-0451
Prakhyat MathurDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0002-3129-4465
Meera PannikkatDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0009-0002-0041-3828
Sophia KrasnyDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.
Rama AldakhlallahDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0009-0008-5833-0244
Alan ChuCalibr-Skaggs Institute for Innovative Medicines, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0002-6669-9649
Kristen A JohnsonCalibr-Skaggs Institute for Innovative Medicines, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0003-0788-7217
Danielle A GrotjahnDepartment of Integrative Structural and Computational Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0001-5908-7882
Timothy E ShuttDepartment of Medical Genetics, Cumming School of Medicine, Hotchkiss Brain Institute, Snyder Institute for Chronic Diseases, Alberta Children's Hospital Research Institute, University of Calgary, Calgary, AB T2N 4N1, Canada.
R Luke WisemanDepartment of Molecular and Cellular Biology, The Scripps Research Institute, La Jolla, CA 92037.ORCID 0000-0001-9287-6840

Funding

IMPACTING MITOCHONDRIAL FUNCTION THROUGH ALTERED PROTEASE ACTIVITYR01NS095892 · NINDS · SCRIPPS RESEARCH INSTITUTE, THE · PI WISEMAN, ROCKLAND LUKE · 2016 to 2025
$5.3M
Defining Endoplasmic Reticulum Stress-Development Mitochondria RemodelingRF1NS125674 · NINDS · SCRIPPS RESEARCH INSTITUTE, THE · PI WISEMAN, ROCKLAND LUKE · 2022 to 2022
$2.4M
Structure-function studies of DELE1-mediated activation of the integrated stress responseR01AG088908 · NIA · SCRIPPS RESEARCH INSTITUTE, THE · PI Rockland Luke Wiseman · 2024 to 2026
$1.9M
Defining Endoplasmic Reticulum Stress-Development Mitochondria RemodelingR01NS125674 · NINDS · SCRIPPS RESEARCH INSTITUTE, THE · PI Rockland Luke Wiseman · 2025 to 2026
$1.4M
American Heart Association (AHA) 0000Canadian Institutes of Health Research (CIHR) 0000George E. Hewitt Foundation for Medical Research (THE GEORGE E HEWITT FOUNDATION FOR MEDICAL RESEARCH) 00000HHS | NIH | National Institute on Aging (NIA) AG088908HHS | NIH (NIH) NS095892HHS | NIH (NIH) NS125674Hotchkiss Brain Institute International Graduate Recruitment Scholarship 0000NIA NIH HHS R01 AG088908NINDS NIH HHS R01 NS095892NINDS NIH HHS R01 NS125674NINDS NIH HHS RF1 NS125674NSF | NSF Graduate Research Fellowship Program (GRFP) 0000Wellcome Trust
6 · The paper itself

Abstract

Pathogenic variants in the mitochondrial outer membrane GTPase MFN2 cause the peripheral neuropathy Charcot-Marie-Tooth type 2A (CMT2A). These mutations can disrupt MFN2-dependent regulation of diverse aspects of mitochondrial biology including organelle morphology, motility, mitochondrial-endoplasmic reticulum (ER) contacts (MERCs), and respiratory chain activity. However, no therapies currently exist to mitigate the mitochondrial dysfunction linked to genetic deficiencies in MFN2. Herein, we performed a drug repurposing screen to identify compounds that selectively activate the integrated stress response (ISR)-the predominant stress-responsive signaling pathway responsible for regulating mitochondrial morphology and function. This screen identified the compounds parogrelil and MBX-2982 as potent and selective activators of the ISR through the OMA1-DELE1-HRI signaling axis. We show that treatment with these compounds promotes adaptive, ISR-dependent remodeling of mitochondrial morphology and protects mitochondria against genetic and chemical insults. Moreover, we show that pharmacologic ISR activation afforded by parogrelil restores mitochondrial tubular morphology, promotes mitochondrial motility, rescues MERCs, and enhances mitochondrial respiration in

Indexed as

Charcot-Marie-Tooth DiseaseDrug RepositioningGTP PhosphohydrolasesMitochondriaMitochondrial ProteinsAnimalsHumansMiceSignal TransductionGTP PhosphohydrolasesMFN2 protein, humanMfn2 protein, mouseMitochondrial Proteinsdrug repurposingintegrated stress responsemitochondrial dysfunction

Identifiers

PMID41289394
PMCPMC12685026

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.