World's First Recipient Of Personalized Gene Editing Treatment Lives

                   CPS1 deficiency, a rare genetic disorder affects one in 1.3 million babies. Only half of babies diagnoses with this disorder make it past the first week. If they do survive that long they would have severe mental and developmental delays and eventually need liver transplants. Their bodies are unable to rid themselves of ammonia, a byproduct of protein metabolism which build up in the blood and crosses into the brain. However, KJ’s parents decided to try something new. In New York Time’s article Baby Is Healed With World’s First Personalized Gene-Editing Treatment, Baby KJ was reported to be the first recipient in the world to receive a custom gene editing treatment. KJ’s treatment was customized so CRISPR, a gene editing tool that finds and cuts specific sequences, found just his single point mutation causing this fatal genetic disorder. Every treatment he receives requires a lower and lower dose. Now over 9 ½ months old and thriving, KJ’s trial opens the door to vast possibilities in gene editing field. 

        What does this mean for others? Parents facing a CPS1 deficiency diagnosis for their child now doesn’t necessarily mean an early end and severely impacted quality of life. Cleveland Clinic estimates there could be as many as 7,000 genetic diseases and disorders that can now be reexamined to find a cure. With living proof this technology works it could potentially be applied to infinites cases and varying severities.