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 ...

Gene therapy drugs have the potential to cure some diseases, but some have a price tag of over a million dollars. Who gets access to them and who doesn't?

Scientists at the Broad Institute of MIT and Harvard, Harvard Medical School, and McLean Hospital have discovered a surprising mechanism by which the inherited genetic mutation known to cause ...

A whole lot more than just one genetic mutation determines whether and how disease develops. lvcandy/DigitalVision Vectors via Getty Images Genetic inheritance may sound straightforward: One gene ...

Google DeepMind's new model deciphers how hidden DNA changes drive disease, predicting gene regulation effects with high ...

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

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

On Wednesday, Google's DeepMind AI research lab announced AlphaGenome, an AI model designed to identify disease markers in large volumes of genetic data. It's badly needed in a field that has produced ...

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 ...

The DNA of Chernobyl cleanup workers showed mutations caused by radiation that were also evident in the genes of their ...