title: AAV-mediated liver-directed gene therapy for Acute Intermittent Porphyria: It is safe but is it effective?
authors.affiliation: Telethon Institute of Genetics and Medicine, Pozzuoli (Naples), Italy; Department of Translational Medicine, Federico II University, Naples, Italy. Electronic address: brunetti@tigem.it.
authors.forename: Nicola
authors.initials: N
authors.lastname: Brunetti-Pierri
authors.affiliation: National Institute for Health Research (NIHR) Birmingham Liver Biomedical Research Unit and Centre for Liver Research, University of Birmingham, Birmingham, UK.
authors.forename: Philip N
authors.initials: PN
authors.lastname: Newsome
mesh_terms: D015316:Genetic Therapy; D006801:Humans; D011163:Hydroxymethylbilane Synthase; D017118:Porphyria, Acute Intermittent
chemical_list: D011163:Hydroxymethylbilane Synthase