Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Exton, Pennsylvania, July 10, 2025 (GLOBE NEWSWIRE) -- Recently released research from Spherix Global Insights provides a timely look at the increasingly competitive and complex treatment landscape ...

One dose of an experimental drug cover offer lifetime treatment for people with high cholesterol, but its long-term safety is ...

A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...