Human Embryonic Stem Cell-Derived Wilson's Disease Model for Screening Drug Efficacy.

ABSTRACT: Human pluripotent stem cells (hPSCs) including human embryonic stem cells (hESCs) and human-induced pluripotent stem cells (hiPSCs) have been extensively studied as an alternative cellular model for recapitulating phenotypic and pathophysiologic characters of human diseases. Particularly, hiPSCs generated from the genetic disease somatic cells could provide a good cellular model to screen potential drugs for treating human genetic disorders. However, the patient-derived cellular model has a limitation when the patient samples bearing genetic mutations are difficult to obtain due to their rarity. Thus, in this study, we explored the potential use of hPSC-derived Wilson's disease model generated without a patient sample to provide an alternative approach for modeling human genetic disease by

SUBMITTER: Kim D 

PROVIDER: S-EPMC7226780 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature