In Vivo Correction of Human Phenylketonuria Variants via Prime Editing and Base Editing: A Gateway to Equitable Treatment of Inborn Errors of Metabolism

Watch a 20-minute webinar exploring groundbreaking research on treating phenylketonuria (PKU) through innovative gene editing techniques, presented by Dr. Xiao Wang from the University of Pennsylvania's Cardiovascular Institute. Learn about the development of personalized base-editing and prime-editing therapies for severe, rare hepatic inborn errors of metabolism (IEMs), with a specific focus on PKU caused by variants in the phenylalanine hydroxylase (PAH) gene. Discover how researchers are addressing the limitations of current treatments like liver transplantation by investigating novel therapeutic approaches using genomic editing technologies. Gain insights into their screening platform for testing editing solutions on various PKU variants in hepatocytes. Healthcare professionals can earn PACE credits through Labroots upon completion of this educational presentation.