ArticleNature communications2024
Cannabidiol ameliorates mitochondrial disease via PPARγ activation in preclinical models.
Article in Nature communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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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.
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Who cites it
18 citing papers in PubMed.
- Accelerating Leigh syndrome drug discovery through deep learning screening in brain organoids.Nature communications · 2026Article
- The dual roles of natural cannabidiol in combating oxidative stress and inflammation: A potential intestinal guardian.Redox biology · 2026Review
- Cannabidiol in Developmental Epilepsy: Organoid-Guided Precision Medicine Across Critical Neurodevelopmental Windows.International journal of molecular sciences · 2026Review
- Pluripotent stem-cell-based screening uncovers sildenafil as a mitochondrial disease therapy.Cell · 2026Article
- Butyrate extends health and lifespan in mice with mitochondrial deficiency.Nature communications · 2026Article
- Cannabidiol exerts antiinflammatory effects but maintains T effector memory cell differentiation in humans.JCI insight · 2026Article
- Cannabidiol suppresses emergency MDSCs generation by disturbing EEF1B2-mediated C/EBPJournal for immunotherapy of cancer · 2026Article
- Comparative analysis of 105 datasets across species and tissues reveals differential transcriptomic responses to cannabinoids THC and CBD.Journal of cannabis research · 2025Article
- Expanding research and care for Leigh syndrome: efforts of a patient-led advocacy organization.Research involvement and engagement · 2025Article
- The potential role of cannabidiol (CBD) in lung cancer therapy: a systematic review of preclinical and clinical evidence.Cancer cell international · 2025Review
- Cannabidiol attenuates epileptic phenotype and increases survival in a mouse model of developmental and epileptic encephalopathy type 1.Epilepsia · 2025Article
- Leigh Syndrome: A Comprehensive Review of the Disease and Present and Future Treatments.Biomedicines · 2025Review
- Streamlined Quantification of Microglial Morphology in Mouse Brains Using 3D Immunofluorescence Analysis.Bio-protocol · 2025Article
- Article
- Preventive beneficial effects of cannabidiol in a reserpine-induced progressive model of parkinsonism.Frontiers in pharmacology · 2025Article
- Buprenorphine and cannabidiol co-administration reduces survival in a mouse model of orthopedic trauma.Frontiers in pharmacology · 2025Article
- Long term exposure to dietary cannabinoids inhibits rapid functional tolerance to ethanol inmicroPublication biology · 2025Article
- Disease models of Leigh syndrome: From yeast to organoids.Journal of inherited metabolic disease · 2024Review
Corrections and comments
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Authors and funding
23 authors.
Funding
Abstract
Mutations in mitochondrial energy-producing genes lead to a heterogeneous group of untreatable disorders known as primary mitochondrial diseases (MD). Leigh syndrome (LS) is the most common pediatric MD and is characterized by progressive neuromuscular affectation and premature death. Here, we show that daily cannabidiol (CBD) administration significantly extends lifespan and ameliorates pathology in two LS mouse models, and improves cellular function in fibroblasts from LS patients. CBD delays motor decline and neurodegenerative signs, improves social deficits and breathing abnormalities, decreases thermally induced seizures, and improves neuropathology in affected brain regions. Mechanistically, we identify peroxisome proliferator-activated receptor gamma (PPARγ) as a key nuclear receptor mediating CBD's beneficial effects, while also providing proof of dysregulated PPARγ expression and activity as a common feature in both mouse neurons and fibroblasts from LS patients. Taken together, our results provide the first evidence for CBD as a potential treatment for LS.
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What Socratic holds
Registered trials
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.