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 and Penn Medicine.
The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase deficiency (CPS1). After spending the first several months of his life in the hospital on a very restrictive diet, KJ received the first dose of his unique therapy in February when he was around six months old.
The treatment was safely administered, and he is now growing and thriving.
The case was detailed this week in a study published by The New…