An <i>In Vitro</i> Whole-Organ Liver Engineering for Testing of Genetic Therapies.

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 express

SUBMITTER: Lorvellec M 

PROVIDER: S-EPMC7708813 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature