The Promises and Perils Of Gene Editing
It would have seemed impossible only a few years ago, but medical researchers have been able to edit a baby’s genes so that he does not suffer from a rare genetic disease. A press release from the Children’s Hospital of Philadelphia explains, “In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine. The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. After spending the first several months of his life in the hospital, on a very restrictive diet, KJ received the first dose of his bespoke therapy in February 2025 between six and seven months of age. The treatment was administered safely, and he is now growing well and thriving.”
There have also been cases of gene editing for sickle cell disease and beta thalassemia. But in KJ’s case this is the first time gene editing was done for one individual which means the editing only applies to his case. For those other diseases, anyone who has them can use the same editing process.
