Researchers have engineered a new class of adenine base editors that reduce unwanted bystander mutations by two to three times compared to the widely used ABE8e, while preserving the editing power ...

The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

A year after receiving the world’s first personalized base-editing therapy for a lethal CPS1 gene mutation, Baby KJ is healthy and has avoided a liver transplant. The unprecedented treatment, ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his life. But the scientific advances that made the groundbreaking treatment ...