Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...

Gene therapies have a lot of potential to improve health outcomes for patients with life-threatening diseases but face a ...

The firm's chief scientific officer called the achievement "a major milestone for Spirovant and for the cystic fibrosis ...

CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...

Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.

Cyclosporin H boosts a new lentivirus-delivered gene therapy with enhanced anti-sickling properties in blood stem cells from ...

A new type of therapy that ‘edits’ a gene in patients with a rare heart condition has been shown to be safe and effective, ...

Delivering very large genes to cells can pose a serious challenge, so scientists developed a new method. | Genetics And ...

William Blair analyst Sami Corwin has maintained their bullish stance on RCKT stock, giving a Buy rating on November 15.Don't Miss our Black ...

By targeting the TTR gene directly in the liver, the therapy “has opened up the door” to permanent treatment, says Sarah ...

Autism-linked SHANK3 gene mutations disrupt not only neurons but also oligodendrocytes, essential for producing myelin, which ...