University of Kentucky researchers have developed a new experimental model that could point the way toward more effective ...

Mutations in a gene associated with Crohn's disease have been found to rob critical immune cells of their ability to switch ...

The trial is a phase 1/2 open-label, ascending-dose study of the safety and efficacy of OPGx-LCA5. Recently, the company ...

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

Nearly 7 million Americans currently live with Alzheimer’s and by 2060, experts say that number could be as high as 14 ...

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

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...

The US FDA has tightened the use of a Duchenne muscular dystrophy gene therapy after two teenage patients died from acute ...

We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...

The two main approaches for discovering disease genes reveal distinct aspects of biology, a new study shows. While both ...

Women have risk factors such as genetics, hormones, and sex-specific responses to stress.