title: Strategies for treating mitochondrial disorders: an update.
abstract: Mitochondrial diseases are a heterogeneous group of disorders resulting from primary dysfunction of the respiratory chain due to both nuclear and mitochondrial DNA mutations. The wide heterogeneity of biochemical dysfunctions and pathogenic mechanisms typical of this group of diseases has hindered therapy trials; therefore, available treatment options remain limited. Therapeutic strategies aimed at increasing mitochondrial functions (by enhancing biogenesis and electron transport chain function), improving the removal of reactive oxygen species and noxious metabolites, modulating aberrant calcium homeostasis and repopulating mitochondrial DNA could potentially restore the respiratory chain dysfunction. The challenge that lies ahead is the translation of some promising laboratory results into safe and effective therapies for patients. In this review we briefly update and discuss the most feasible therapeutic approaches for mitochondrial diseases.
authors.affiliation: Section of Neurology, Department of Neurological and Movement Sciences, University of Verona, Verona, Italy.
authors.forename: Mauro
authors.initials: M
authors.lastname: Scarpelli
authors.affiliation: Clinical Neurology, Section for Neuromuscular Diseases and Neuropathies, University Hospital "Spedali Civili", Brescia, Italy.
authors.forename: Alice
authors.initials: A
authors.lastname: Todeschini
authors.affiliation: Clinical Neurology, Section for Neuromuscular Diseases and Neuropathies, University Hospital "Spedali Civili", Brescia, Italy.
authors.forename: Fabrizio
authors.initials: F
authors.lastname: Rinaldi
authors.affiliation: Clinical Neurology, Section for Neuromuscular Diseases and Neuropathies, University Hospital "Spedali Civili", Brescia, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Rota
authors.affiliation: Clinical Neurology, Section for Neuromuscular Diseases and Neuropathies, University Hospital "Spedali Civili", Brescia, Italy.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Padovani
authors.affiliation: Clinical Neurology, Section for Neuromuscular Diseases and Neuropathies, University Hospital "Spedali Civili", Brescia, Italy. Electronic address: massimiliano.filosto@unibs.it.
authors.forename: Massimiliano
authors.initials: M
authors.lastname: Filosto
mesh_terms: D002118:Calcium / Q000378:metabolism; D004272:DNA, Mitochondrial / Q000235:genetics / Q000627:therapeutic use*; D004579:Electron Transport / Q000235:genetics; D006706:Homeostasis; D006801:Humans; D008928:Mitochondria / Q000378:metabolism*; D028361:Mitochondrial Diseases / Q000378:metabolism / Q000628:therapy*; D063269:Mitochondrial Turnover; D017382:Reactive Oxygen Species / Q000378:metabolism
chemical_list: D004272:DNA, Mitochondrial; D017382:Reactive Oxygen Species; D002118:Calcium
keywords: Mitochondrial DNA; Mitochondrial diseases; Respiratory chain; Therapy