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Gene Ther. 2017 May;24(5):263-264. doi: 10.1038/gt.2016.90. Epub 2017 Jan 12.

Delivering efficient liver-directed AAV-mediated gene therapy.

Author information

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.
PMID:
28079048
DOI:
10.1038/gt.2016.90
[Indexed for MEDLINE]

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