A baby born with a rare and devastating genetic condition has become the first person ever to be successfully treated with a personalized CRISPR therapy. After receiving three doses of the therapy in the past few months, the infant is now 9.5 months old and thriving, his doctors report.
"We want each and every patient to have the potential to experience the same results we saw in this first patient," Dr. Kiran Musunuru, a professor for translational research at the University of Pennsylvania's Perelman School of Medicine, said in a statement. "The promise of gene therapy that we've heard about for decades is coming to fruition, and it's going to utterly transform the way we approach medicine."
