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

Studies of Hunter and Hurler syndromes on hold ...

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

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

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.

U.S. drugmaker Eli Lilly and Company signed an agreement worth up to $1.12 billion with Seamless Therapeutics, the ...

Deepwater's Gene Munster joins 'Fast Money' to talk the sell-off in software stocks, Apple quarterly results, and more.

Sometimes, in genetics, two wrongs do make a right. A research team has recently shown that two harmful genetic variants, ...

The study, published in Genomic Psychiatry, identified how stress hormones activate specific RNA molecules called long ...

Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers ...