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

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

Although heart cells and skin cells contain identical instructions for creating proteins encoded in their DNA, they're able ...

An experimental gene-editing treatment shows promise for permanently lowering levels of cholesterol and triglycerides, ...

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

Boosting levels of a certain molecule that declines with age was found to restore memory and brain function in Alzheimer’s ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. The baby, KJ Muldoon of Clifton Heights, ...

Alzheimer's Disease (AD), the leading cause of dementia, affects nearly 40 million individuals globally, resulting in a ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

Subscribe to our newsletter for the latest sci-tech news updates. Published in Nature Communications, scientists from the Wellcome Sanger Institute reveal insights into how our genes influence the ...