title: Read-through therapy for mitochondrial DNA nonsense mutations.
abstract: Disorders resulting from mitochondrial DNA (mtDNA) mutations, including nonsense mutations, do not yet have causal treatments. As we discuss here, read-through therapies appear to be a promising approach to the treatment of disorders arising from nuclear DNA (nDNA) nonsense mutations. The genetics of mitochondrial DNA suggest that this therapy will also be successful in the treatment of mitochondriopathies.
authors.affiliation: Departamento de Bioquímica, Biología Molecular y Celular, Centro de Investigaciones Biomédicas En Red de Enfermedades Raras (CIBERER), Universidad de Zaragoza, Spain.
authors.forename: David
authors.initials: D
authors.lastname: Pacheu-Grau
authors.forename: Aurora
authors.initials: A
authors.lastname: Gómez-Durán
authors.forename: Ester
authors.initials: E
authors.lastname: López-Gallardo
authors.forename: Julio
authors.initials: J
authors.lastname: Montoya
authors.forename: Eduardo
authors.initials: E
authors.lastname: Ruiz-Pesini
mesh_terms: D018389:Codon, Nonsense; D004272:DNA, Mitochondrial / Q000235:genetics*; D006801:Humans; D028361:Mitochondrial Diseases / Q000235:genetics / Q000628:therapy*
chemical_list: D018389:Codon, Nonsense; D004272:DNA, Mitochondrial