Children born with a specific form of genetic deafness now have a treatment option that did not exist six weeks ago. On April ...

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant ...

Children and adults born with severe hearing loss caused by mutations in the OTOF gene now have a treatment option that did not exist two months ago. On April 23, 2026, the FDA granted accelerated ...

One of the more exciting opportunities in medical technology is using a virus to carry genetic information in the form of DNA and RNA in the cells. One of the most commonly used viruses is ...

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 ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...

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 ...

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 ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.