A Medical First: Personalized Gene Therapy Saves Infant with Rare Disorder

    The University of Pennsylvania and The Children’s Hospital of Philadelphia (CHOP) have recently made a outstanding discovery that used ‘custom- built gene therapy’ to treat a baby with a rare and deadly condition called CPS1 deficiency. This is a genetic deficiency that creates a high amount of ammonia in your blood which can be toxic especially to the brain. With help from the National Institute of Health, the team used CRISPR gene-editing technology to fix this error in the baby’s liver cells. This marks the first CRISPR therapy treatment that can hopefully show a glimpse into the future about how other rare diseases can be treated.