Primary Mitochondrial Diseases
Åsander Frostner E, Simón Serrano S, Chamkha I, Donnelly E, Elmér E, Hansson MJ
Towards a treatment for mitochondrial disease: current compounds in clinical development. Bioenerg Commun 2022.4. https://doi.org/10.26124/bec:2022-0004.
Chinnery PF.
Mitochondrial Disorders Overview. 2000 Jun 8 [Updated 2014 Aug 14]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2018. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1224/.
Russell OM, Gorman GS, Lightowlers RN, Turnbull DM.
Mitochondrial Diseases: Hope for the Future. Cell. 2020 Apr 2;181(1):168-188. doi: 10.1016/j.cell.2020.02.051. Epub 2020 Mar 26. PMID: 32220313.
Gorman GS et al.
Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease. Ann Neurol. 2015;77(5):753-759. doi:10.1002/ana.24362 Epub 2015 Mar 28. PMID: 25652200; PMCID: PMC4737121.
McElroy GS et al.
NAD+ Regeneration Rescues Lifespan, but Not Ataxia, in a Mouse Model of Brain Mitochondrial Complex I Dysfunction. Cell Metab. 2020 Aug 4;32(2):301-308.e6. doi: 10.1016/j.cmet.2020.06.003. Epub 2020 Jun 22. PMID: 32574562; PMCID: PMC7415718.
Pirinen E et al.
Niacin Cures Systemic NAD+ Deficiency and Improves Muscle Performance in Adult-Onset Mitochondrial Myopathy. Cell Metab. 2020 Jun 2;31(6):1078-1090.e5. doi: 10.1016/j.cmet.2020.04.008. Epub 2020 May 7. Erratum in: Cell Metab. 2020 Jul 7;32(1):144. PMID: 32386566.
Titov DV, Cracan V, Goodman RP, Peng J, Grabarek Z, Mootha VK.
Complementation of mitochondrial electron transport chain by manipulation of the NAD+/NADH ratio. Science. 2016 Apr 8;352(6282):231-5. doi: 10.1126/science.aad4017. Epub 2016 Apr 7. PMID: 27124460; PMCID: PMC4850741.
Seo KS et al.
KL1333, a Novel NAD+ Modulator, Improves Energy Metabolism and Mitochondrial Dysfunction in MELAS Fibroblasts. Front Neurol. 2018 Jul 5;9:552. doi: 10.3389/fneur.2018.00552. eCollection 2018. PubMed PMID: 30026729; PubMed Central PMCID: PMC6041391.
Ehinger JK et al.
Cell-permeable succinate prodrugs bypass mitochondrial complex I deficiency. Nat Commun. 2016 Aug 9;7:12317. doi: 10.1038/ncomms12317. PubMed PMID: 27502960; PubMed Central PMCID: PMC4980488.
Bakare AB, Rao RR, Iyer S.
Cell-Permeable Succinate Increases Mitochondrial Membrane Potential and Glycolysis in Leigh Syndrome Patient Fibroblasts. Cells. 2021 Aug 31;10(9):2255. doi: 10.3390/cells10092255. PMID: 34571904; PMCID: PMC8470843.
Thorburn DR, Rahman J, Rahman S.
Mitochondrial DNA-Associated Leigh Syndrome and NARP. 2003 Oct 30 [Updated 2017 Sep 28]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2018. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1173/.
Traumatic Brain Injury
Hansson MJ, Elmér E.
Cyclosporine as Therapy for Traumatic Brain Injury. Neurotherapeutics. 2023 Aug 10. doi: 10.1007/s13311-023-01414-z. Epub ahead of print. PMID: 37561274.
Kelsen J et al.
Copenhagen Head Injury Ciclosporin (CHIC) study: A phase IIa safety, pharmacokinetics and biomarker study of ciclosporin in severe head injury patients. J Neurotrauma. 2019 Jun 18. doi: 10.1089/neu.2018.6369. [Epub ahead of print] PMID: 31210099.
Karlsson M et al.
Neuroprotective Effects of Cyclosporine in a Porcine Pre-Clinical Trial of Focal Traumatic Brain Injury. J Neurotrauma. 2018 Jul 24. doi: 10.1089/neu.2018.5706. [Epub ahead of print] PMID: 29929438.
Maas AIR et al.
Traumatic brain injury: integrated approaches to improve prevention, clinical care, and research. Lancet Neurol. 2017 Dec;16(12):987-1048. doi: 10.1016/S1474-4422(17)30371-X. Epub 2017 Nov 6. Review. PubMed PMID: 29122524.
Cyclophilin inhibitors
Simón Serrano S et al.
Synergistic Effects of Sanglifehrin-Based Cyclophilin Inhibitor NV651 with Cisplatin in Hepatocellular Carcinoma. Cancers (Basel). 2022 Sep 20;14(19):4553. doi: 10.3390/cancers14194553. PMID: 36230472; PMCID: PMC9559492.
Simón Serrano S et al.
Novel Cyclophilin Inhibitor Decreases Cell Proliferation and Tumor Growth in Models of Hepatocellular Carcinoma. Cancers (Basel). 2021 Jun 18;13(12):3041. doi: 10.3390/cancers13123041. PMID: 34207224; PMCID: PMC8234462.
Simón Serrano S et al.
Evaluation of NV556, a Novel Cyclophilin Inhibitor, as a Potential Antifibrotic Compound for Liver Fibrosis. Cells. 2019 Nov 8;8(11). pii: E1409. doi: 10.3390/cells8111409. PMID: 31717385.
Kuo J et al.
Cyclophilin Inhibitor NV556 Reduces Fibrosis and Hepatocellular Carcinoma Development in Mice With Non-Alcoholic Steatohepatitis. Front Pharmacol. 2019 Sep 26;10:1129. doi: 10.3389/fphar.2019.01129. eCollection 2019. PMID: 31611801.
Other
Piel S et al.
Succinate prodrugs as treatment for acute metabolic crisis during fluoroacetate intoxication in the rat. Mol Cell Biochem. 2022 Oct 25. doi: 10.1007/s11010-022-04589-9. Epub ahead of print. PMID: 36282352.