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

From revolutionary CRISPR treatment to heavy drinking dangers, here's what you need to know about the latest health research.

Researchers at Tel Aviv University led a large-scale international study that opens a new avenue for treating the fatal ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

N EW ORLEANS – A single injection of a gene-edited protein was deemed productive and safe in patients with dyslipidemia that ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

Opus Genetics has completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the US Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber ...

The agency is establishing a network of care centers to expand access to regenerative medicine beyond regions served by major ...