New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

These results are what we hoped for when we first began developing this approach,' one researcher said. View on euronews ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...

In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...

The MCO-010 vector uses surviving cells in the retina to produce a photosensitive protein that may help patients regain some ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...