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An In Vitro Whole-Organ Liver Engineering for Testing of Genetic Therapies

Lorvellec, M; Pellegata, AF; Maestri, A; Turchetta, C; Alvarez Mediavilla, E; Shibuya, S; Jones, B; ... Gissen, P; + view all (2020) An In Vitro Whole-Organ Liver Engineering for Testing of Genetic Therapies. iScience , 23 (12) , Article 101808. 10.1016/j.isci.2020.101808. Green open access

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Abstract

Explosion of gene therapy approaches for treating rare monogenic and common liver disorders created an urgent need for disease models able to replicate human liver cellular environment. Available models lack 3D liver structure or are unable to survive in long-term culture. We aimed to generate and test a 3D culture system that allows long-term maintenance of human liver cell characteristics. The in vitro whole-organ "Bioreactor grown Artificial Liver Model" (BALM) employs a custom-designed bioreactor for long-term 3D culture of human induced pluripotent stem cells-derived hepatocyte-like cells (hiHEPs) in a mouse decellularized liver scaffold. Adeno-associated viral (AAV) and lentiviral (LV) vectors were introduced by intravascular injection. Substantial AAV and LV transgene expression in the BALM-grown hiHEPs was detected. Measurement of secreted proteins in the media allowed non-invasive monitoring of the system. We demonstrated that humanized whole-organ BALM is a valuable tool to generate pre-clinical data for investigational medicinal products.

Type: Article
Title: An In Vitro Whole-Organ Liver Engineering for Testing of Genetic Therapies
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.isci.2020.101808
Publisher version: http://dx.doi.org/10.1016/j.isci.2020.101808
Language: English
Additional information: © 2020 The Authors. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)
Keywords: Bioengineering, Clinical Genetics, Tissue Engineering
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Surgery and Interventional Sci
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Biology and Cancer Dept
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10117669
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