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

A Google DeepMind invention that uses artificial intelligence (AI) to predict how DNA mutations behave could have a ...

A small change to a powerful molecular tool — using the CRISPR-Cas13 enzyme, which can detect RNA from viruses and cells — ...

Fixing gene defects with designer DNA is on the horizon after Google DeepMind launched a pioneering new AI tool.

What if doctors could look at a person’s DNA and not only spot a harmful mutation, but also predict the kind of disease it may cause—before symptoms fully appear? Scientists at the Icahn School of ...

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

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

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 ...

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 ...

Only around two percent of the human genome codes for proteins, and while those proteins carry out many important functions of the cell, the rest of the genome cannot be ignored. However, for decades ...

Affected populations: POLG-related diseases are a group of rare, incurable conditions caused by inherited gene mutations. These disease-causing mutations are carried by up to 2% of people of Northern ...