Out of every 60,000 births, a baby arrives to face the world without a fully functioning immune system leaving them unequipped to fight even the most common infections. Children with this rare life-threatening genetic condition, known as severe combined immunodeficiency (SCID), have the best chance at a healthy future if they undergo a stem cell transplant before they are 3 ½ months old.
Seattle Children’s recently opened a clinical trial that is seeking a potentially safer, less aggressive and equally effective path to a cure by using a novel gene therapy to fix the faulty gene that causes the most common type of SCID.
On the Pulse met with the trial’s principal investigator, Dr. Aleksandra Petrovic, a pediatric transplant specialist and researcher at Seattle Children’s Research Institute’s Center for Immunity and Immunotherapies, to learn more about the experimental therapy available through this trial.
