
In February, a team of scientists achieved a long-held scientific goal — creating a bespoke CRISPR editing therapy to treat a single patient with a unique genetic mutation. “Baby KJ” got his own custom treatment only six months after he was born. Now, Rebecca Ahrens-Nicklas, a pediatrician and gene therapy scientist at CHOP, and Kiran Musunuru, a UPenn gene editing scientist, have a plan to replicate that success. They’re joining Post-Hoc Live to talk about what’s next.