title: Delivering efficient liver-directed AAV-mediated gene therapy.
authors.affiliation: Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health, University College London, London, UK.|Department of Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.|Maternal and Fetal Medicine, Gene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.
authors.forename: J
authors.initials: J
authors.lastname: Baruteau
authors.affiliation: Maternal and Fetal Medicine, Gene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.|Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg, South Africa.
authors.forename: S N
authors.initials: SN
authors.lastname: Waddington
authors.affiliation: Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Sydney, New South Wales, Australia.|Discipline of Child and Adolescent Health, The University of Sydney, Sydney, New South Wales, Australia.
authors.forename: I E
authors.initials: IE
authors.lastname: Alexander
authors.affiliation: Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health, University College London, London, UK.|Department of Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.|MRC Laboratory for Molecular Biology, University College London, London, UK.
authors.forename: P
authors.identifier: 0000-0002-9712-6122
authors.initials: P
authors.lastname: Gissen
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D008099:Liver / Q000378:metabolism / Q000502:physiology; D023421:Models, Animal