Delivering efficient liver-directed AAV-mediated gene therapy

Affiliations

• ¹ Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health, University College London, London, UK.
• ² Department of Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
• ³ Maternal and Fetal Medicine, Gene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.
• ⁴ Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg, South Africa.
• ⁵ Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Sydney, New South Wales, Australia.
• ⁶ Discipline of Child and Adolescent Health, The University of Sydney, Sydney, New South Wales, Australia.
• ⁷ MRC Laboratory for Molecular Biology, University College London, London, UK.