Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

LONDON, Jan 31 (Reuters) - Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care ...