The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

KJ Muldoon, the first infant to undergo gene-editing therapy, has accomplished another first as a 1-year-old — his first steps KJ is walking at home ahead of Christmas after being hospitalized last ...

A year ago, doctors did not know if KJ Muldoon would survive infancy. Today, he is learning to walk at home with his family. His first steps follow a medical first that changed the course of his life.

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...