title: Epigenetic therapy reprograms hereditary disease.
abstract: In this issue of Blood, Makarona et al demonstrate that histone deacetylase (HDAC) inhibitors (HDACis) in glucose-6-phosphate dehydrogenase (G6PD)-deficient cells reinstates enzyme activity by boosting gene transcription. This therapeutic approach opens new avenues for preclinical and clinical studies to treat not only chronic nonspherocytic hemolytic anemia caused by severe G6PD variants, but also other genetic diseases.
authors.affiliation: COMPLUTENSE UNIVERSITY OF MADRID.
authors.forename: José M
authors.identifier: http://orcid.org/0000-0001-8926-881X
authors.initials: JM
authors.lastname: Bautista
mesh_terms: D044127:Epigenesis, Genetic / Q000187:drug effects*; D005954:Glucosephosphate Dehydrogenase / Q000096:biosynthesis* / Q000235:genetics*; D005955:Glucosephosphate Dehydrogenase Deficiency / Q000235:genetics*; D056572:Histone Deacetylase Inhibitors / Q000494:pharmacology*; D006801:Humans; D014158:Transcription, Genetic / Q000187:drug effects*
chemical_list: D056572:Histone Deacetylase Inhibitors; D005954:Glucosephosphate Dehydrogenase