Delivering efficient liver-directed AAV-mediated gene therapy
Epub 2017 Jan 12.
S N Waddington
I E Alexander
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.
Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg, South Africa.
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.
MRC Laboratory for Molecular Biology, University College London, London, UK.
No abstract available
Research Support, Non-U.S. Gov't
Dependovirus / genetics*
Genetic Therapy / methods*
Genetic Vectors / therapeutic use*
Liver / metabolism
Liver / physiology
MR/N019075/1/MRC_/Medical Research Council/United Kingdom