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

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

Cardiovascular disease remains the foremost cause of morbidity and mortality globally, affecting millions of individuals. Recent discoveries illuminate the substantial role of genetics in ...

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

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

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

The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency. It ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

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

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

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

A new type of therapy that 'edits' a gene in patients with a rare heart condition has been shown to be safe and effective, according to research from UCL and the Royal Free Hospital. Results for the ...