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

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

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

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

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

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

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

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