title: Gene therapy for inherited diseases of liver metabolism.
abstract: Gene therapy is entering the stage of initial clinical development to treat a growing number of inherited metabolic diseases. This review outlines the development of liver-directed gene therapy for diseases caused by deficiencies of enzymes that are primarily expressed in the liver and discusses the disorders that appear most promising for clinical translation.
authors.affiliation: 1 Telethon Institute of Genetics and Medicine , Pozzuoli, Naples 80078, Italy .
authors.forename: Pasquale
authors.initials: P
authors.lastname: Piccolo
authors.forename: Nicola
authors.initials: N
authors.lastname: Brunetti-Pierri
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008107:Liver Diseases / Q000235:genetics / Q000628:therapy*; D008659:Metabolic Diseases / Q000235:genetics / Q000628:therapy*