Summary
PROJECT SUMMARY Inborn errors of metabolism (IEMs) are rare, devasting disorders arising from pathogenic variants in genes encoding enzymes of key biochemical pathways. The liver plays an important role in the pathogenesis of over 150 IEMs, often failing to metabolize a toxic metabolite that can injure secondary organs, such as brain. Liver transplantation is employed in some IEMs; however, its utility is limited by scarcity of donors and lifelong risk of post-transplant complications. To address the unmet medical need of IEM patients, we aim to develop a master protocol for the rapid developm
