Principle Investigators: Vivian Hwa, PhD, and Takanori Takebe, MD
Recent progress in developing “mini” human liver organs (liver “organoids”) from human cells offer exciting model systems for studying human liver diseases and for potential therapeutic investigations. The normal human liver is critically important for responding to endocrine hormonal cues, such as growth hormone (GH), which regulates the production of IGF-I (insulin-like growth factor I), important for normal human growth, and hormones such as insulin, which regulates blood glucose levels. The importance of appropriate responsiveness to these hormonal cues is highlighted by the many genetic defects we (and others) have identified in GH insensitive children who suffer from severe growth failure, often accompanied by metabolic problems that complicate diagnosis and raise questions of how best to treat the affected child.
Based on our extensive experience with human cell-derived liver organoids, together with our unique collection of patient cells carrying growth disorder genetic defects, we will assess the clinical utility of patient cell-derived liver organoids for evaluating individual therapeutic options, towards improving patient management and care.