Your browser doesn't support javascript.
loading
An In Vitro Whole-Organ Liver Engineering for Testing of Genetic Therapies.
Lorvellec, Maëlle; Pellegata, Alessandro Filippo; Maestri, Alice; Turchetta, Chiara; Alvarez Mediavilla, Elena; Shibuya, Soichi; Jones, Brendan; Scottoni, Federico; Perocheau, Dany P; Cozmescu, Andrei Claudiu; Delhove, Juliette M; Kysh, Daniel; Gjinovci, Asllan; Counsell, John R; Heywood, Wendy E; Mills, Kevin; McKay, Tristan R; De Coppi, Paolo; Gissen, Paul.
Afiliação
  • Lorvellec M; MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK.
  • Pellegata AF; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Maestri A; Developmental Biology and Cancer Research & Teaching Department, Stem Cells & Regenerative Medicine Section, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Turchetta C; MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK.
  • Alvarez Mediavilla E; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Shibuya S; Department of Chemistry, Materials and Chemical Engineering "Giulio Natta," Politecnico di Milano, Milan 20133, Italy.
  • Jones B; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Scottoni F; Developmental Biology and Cancer Research & Teaching Department, Stem Cells & Regenerative Medicine Section, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Perocheau DP; Developmental Biology and Cancer Research & Teaching Department, Stem Cells & Regenerative Medicine Section, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Cozmescu AC; Developmental Biology and Cancer Research & Teaching Department, Stem Cells & Regenerative Medicine Section, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Delhove JM; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Kysh D; MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK.
  • Gjinovci A; NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London, London WC1N 1EH, UK.
  • Counsell JR; Robinson Research Institute, University of Adelaide, Adelaide, SA, 5006, Australia.
  • Heywood WE; MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK.
  • Mills K; MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK.
  • McKay TR; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.
  • De Coppi P; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  • Gissen P; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
iScience ; 23(12): 101808, 2020 Dec 18.
Article em En | MEDLINE | ID: mdl-33305175
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.
Palavras-chave
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: IScience Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Reino Unido
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: IScience Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Reino Unido