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 ...

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 ...

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 ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Today, after receiving a gene therapy developed at the University of California San Diego, Natalie is thriving at 34 years ...

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

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 ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

The genetic treatment targeted a particular kind of congenital deafness and will soon be tried in children who are younger. By Gina Kolata Gina Kolata visited the Children’s Hospital of Philadelphia ...

Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these ...