Gene therapy

Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare inherited eye diseases

This year’s winners include hundreds of physicists across more than 30 institutions

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the approach

Fresh treatments for rare skin diseases shift the focus from symptom management to repair and help children with such conditions live active lives

You have around 35 trillion red blood cells moving around your body at all times. Typically they are rounded and flexible. What happens when they aren’t?

Here’s the best, most inspiring and coolest science we encountered this year

From new uses for weight-loss drugs to the first CRISPR gene editing therapy, these were some of the most impactful health stories of the year

Most people with sickle cell disease who received a new gene editing treatment saw their pain resolve for at least one year, but longer follow up is needed

A newly approved CRISPR therapy could transform the treatment of sickle cell disease and beta-thalassemia—but the technology is expensive

A new book argues that genomics is overshadowing environmental and social solutions to better health for all

The treatment will be reviewed by an outside panel this week

The gene therapy could save kids with severe combined immune deficiency