Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

Virginia Tech researchers discovered an indication hidden in the brain that may help doctors identify children suffering from ...

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

Researchers led by the University of Vienna and Liège University Hospital Center have identified genetic variants associated ...

A viral infection can trigger a neurological disease similar to amyotrophic lateral sclerosis (ALS) in mice with a specific ...

Quince Therapeutics’ novel method of delivering steroids in a patient’s own red blood cells has turned out to be a bust in ataxia-telangiectasia (A-T), a rare inherited disorder. | Quince Therapeutics ...

Virginia Tech researchers have discovered an indication hidden in the brain that may help doctors identify children suffering ...

When two defective gene variants combine, normal protein function can sometimes be restored.

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only minor ...

ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...

People often worry they will get Alzheimer’s disease if their parents did. But engaging in a healthy lifestyle can outweigh ...