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

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

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 ...

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

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct gene-editing experiments. The device, CRISPR-GPT, is an artificial intelligence lab ...

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 ...

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

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 ...