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

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

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

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

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

Virginia Tech researchers discovered an indication hidden in the brain that may help doctors identify children suffering from ...

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

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

Baylor College of Medicine researchers are part of a collaborative research group with AstraZeneca and Memorial Sloan ...

A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...

Studies of Hunter and Hurler syndromes on hold ...

Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for a progressive muscle‑wasting disorder showed sustained benefit in ...