Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...

The immune system faces a delicate balancing act: it must be aggressive enough to fight infections and cancer, yet restrained ...

Centenary Institute researchers have uncovered new genetic causes of inherited heart conditions, providing families with ...

Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...

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

Seed plants are essential as a source of food, fuel, medicine, and more. Now, a multidisciplinary team of researchers has ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

Dangerously high levels of “bad” cholesterol and triglycerides may be reduced by gene editing, in what study researchers hope will be a “one and done” treatment.