Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

In the 35 years since the first gene-therapy trial for a rare genetic disease, the field has advanced to new techniques and to more diseases. That first patient, 4-year-old Ashanthi DeSilva, largely ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...