Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

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

A new AI model from Google DeepMind called AlphaGenome can predict how DNA mutations affect biological processes, helping pinpoint genes linked to diseases and speeding up drug discovery.

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

Euan Ashley's lab explores the intricate interactions of gene variants. Tiny "typos," or genetic mutations, can sneak into ...

Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common ...

Google DeepMind has introduced AlphaGenome, an AI tool that analyses large sections of DNA to predict how genetic mutations ...

No gene acts alone: interacting variants and protein partnerships can worsen, mask or even rescue disease risk, demanding ...

Once disregarded as “junk DNA,” scientists now know that this molecular dark matter is crucial for determining gene activity ...

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

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