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

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

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

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

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

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem ...

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...

Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.

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