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 ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine One of the more exciting opportunities in medical ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...