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

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

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

Scientists achieved a genetic breakthrough by removing an extra human chromosome using gene-editing technology. This landmark study successfully deleted the surplus chromosome 21 responsible for Down ...

Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...

Studies of Hunter and Hurler syndromes on hold ...

ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

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

By Kamal Choudhury Jan 27 (Reuters) - Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...