title: Exploiting the unique regenerative capacity of the liver to underpin cell and gene therapy strategies for genetic and acquired liver disease.
abstract: The number of genetic or acquired diseases of the liver treatable by organ transplantation is ever-increasing as transplantation techniques improve placing additional demands on an already limited organ supply. While cell and gene therapies are distinctly different modalities, they offer a synergistic alternative to organ transplant due to distinct architectural and physiological properties of the liver. The hepatic blood supply and fenestrated endothelial system affords relatively facile accessibility for cell and/or gene delivery. More importantly, however, the remarkable capacity of hepatocytes to proliferate and repopulate the liver creates opportunities for new treatments based on emerging technologies. This review will summarise current understanding of liver regeneration, describe clinical and experimental cell and gene therapeutic modalities and discuss critical challenges to translate these new technologies to wider clinical utility. This article is part of a Directed Issue entitled: "Regenerative Medicine: the challenge of translation".
authors.affiliation: Gene Therapy Research Unit of The Children's Medical Research Institute and The Children's Hospital at Westmead, Australia.
authors.forename: Grant J
authors.initials: GJ
authors.lastname: Logan
authors.affiliation: Gene Therapy Research Unit of The Children's Medical Research Institute and The Children's Hospital at Westmead, Australia.
authors.forename: Gustavo
authors.initials: G
authors.lastname: de Alencastro
authors.affiliation: Gene Therapy Research Unit of The Children's Medical Research Institute and The Children's Hospital at Westmead, Australia; University of Sydney Discipline of Paediatrics and Child Health, Westmead, NSW 2145, Australia.
authors.forename: Ian E
authors.initials: IE
authors.lastname: Alexander
authors.affiliation: The Centre for Medical Research, Harry Perkins Institute of Medical Research, Crawley, WA 6009, Australia. Electronic address: george.yeoh@uwa.edu.au.
authors.forename: George C
authors.initials: GC
authors.lastname: Yeoh
mesh_terms: D000818:Animals; D049109:Cell Proliferation; D015316:Genetic Therapy / Q000379:methods; D022781:Hepatocytes / Q000166:cytology*; D006801:Humans; D008099:Liver / Q000166:cytology* / Q000502:physiology*; D008107:Liver Diseases / Q000235:genetics / Q000601:surgery / Q000628:therapy; D008115:Liver Regeneration / Q000502:physiology*; D016031:Liver Transplantation / Q000379:methods; D044968:Regenerative Medicine / Q000379:methods / Q000639:trends
keywords: Gene therapy; Liver; Metabolic disease; Regeneration