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

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 ...

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

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

The Influenza A virus (IAV) has been the cause of six major flu pandemics, responsible for 50 to 100 million deaths globally.

Key takeawaysA blood stem cell gene therapy co-developed by UCLA's Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare ...

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 ...

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

Abeona Therapeutics Inc. (Nasdaq: ABEO), a commercial-stage biopharmaceutical company developing cell and gene therapies for ...