“We’re still very much in the early stages,” a gene therapy expert said. “But every day, he’s showing us signs that he’s growing and thriving.”

Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a blueprint for potentially life-saving, gene-editing…

Il piccolo era affatto da una rara malattia metabolica ed è stato trattato al Children's Hospital di Philadelphia