It has not been possible to directly investigate the human hepatic GH-IGF-I growth axis. We are currently assessing the utility of human iPSC-derived liver organoid model system (developed by Takanori Takebe’s group) for responsiveness to endocrine hormonal cues such as GH, leveraging our panel of human cells carrying characterized mutations along the GH-IGF-I growth axis. This new model system will permit patient-based, individualized functional evaluation of identified mutations and variants of unknown significance associated with IGF-I deficiency and impaired linear growth, with the future potential for individualized therapy evaluations.
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Cincinnati Children's Hospital Medical Center: Center of Pediatric Genomic (CpG) Award V.Hwa & T.Takebe (Co-PI) Duration: 7/1/17 – 6/30/18
Title: Organoid models of endocrine growth disorder for personalized therapy testing
Goal: to assess the clinical utility of patient-based induced-PSC (iPSC) derived liver organoids for personalized therapeutic testing when abnormal hormonal responses are consequences of genetic defects