A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

Doug Whitney, 76, carries a rare PSEN2 genetic mutation that causes early-onset Alzheimer's but shows no cognitive decline ...

Your DNA contains millions of genetic variants that interact with each other in ways that affect whether diseases such as ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

An experimental gene therapy has slowed the progression of Huntington’s disease by 75 percent, according to results reported ...

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

There is no cure for Huntington's disease, but a new gene treatment is the first to significantly slow the symptoms in human ...

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious ...

Scientists have identified how a gene drives cell dysfunction seen in people with type 2 diabetes, in findings that could ...