The brain penetrant PPARγ agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy
Abstract
Leriglitazone reduces pathologically activated microglia and protects neurons, astrocytes, and oligodendrocytes in experimental models of X-ALD.
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At the highest dose, leriglitazone reduced axonal degeneration and microglia activation in the white matter of the spinal cord and improved motor performance of Abcd1:Abcd2 double KO mice.
“At the highest dose, leriglitazone reduced axonal degeneration and microglia activation in the white matter of the spinal cord. Moreover, leriglitazone was able to improve the motor performance of the mice.”
From Revisiting the Pathogenesis of X-Linked Adrenoleukodystrophy (2025) · cites this paper as [456]
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References 69 · Cited by 40
- Authors
- Rodríguez-Pascau, L., Vilalta, A., Cerrada, M., Traver, E., Forss-Petter, S., Weinhofer, I., Bauer, J., Kemp, S., Pina, G., Pascual, S., Meya, U., Musolino, P. L., Berger, J., Martinell, M., & Pizcueta, P.
- Affiliations
- Minoryx Therapeutics S.L., Barcelona 08302, Spain.
- Department of Pathobiology of the Nervous System, Center for Brain Research, Medical University of Vienna, Vienna 1090, Austria.
- Medical University of Vienna (AT)
- Amsterdam UMC Location University of Amsterdam (NL)
- Massachusetts General Hospital (US)
- Corresponding author
- Pizcueta, Pilar
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2021-06-02
- Volume
- 13
- Issue
- 596
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W3165292144
- Cited by
- 40
- References
- 69
- Citations by year
- 2026: 3 · 2025: 5 · 2024: 8 · 2023: 13 · 2022: 8 · 2021: 3
- FWCI
- 5.62
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- Pure Amsterdam UMC (submittedVersion)
- Funders
- Horizon 2020 Framework Programme (712949)
- Generalitat de Catalunya (RTC-2017-5867-1)
- Politechnika Poznanska (PTQ-13-06015)
- CDTI (IDI-20180106)
- European Union’s Horizon 2020 research and innovation programme under the Marie Skłodowska-Curie grant (712949)
- Nuclis (RD14-1-O114, IDI-20180106)
- Enisa Jóvenes Emprendedores (2012)
- Torres Quevedo (PTQ-13-06015)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Health Sciences › Medicine › Pathology and Forensic Medicine › Multiple Sclerosis Research Studies
- Keywords
- X-linked adrenoleukodystrophy, PPARγ agonists, adrenomyeloneuropathy, cerebral adrenoleukodystrophy, neuroinflammation, oxidative stress, mitochondrial function, very long chain fatty acids, remyelination, oligodendrocytes, myelin debris clearance, multiple sclerosis, experimental autoimmune encephalitis, monocyte-derived macrophages, blood-brain barrier, neuroprotection, adiponectin, target engagement
- MeSH terms
- Adrenoleukodystrophy, Brain, Humans, Oligodendroglia, Endothelial Cells, PPAR gamma
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