Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...

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

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

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 ...

While interested in CAR-T therapies’ potential to achieve durable, drug-free remission in serious autoimmune conditions, the ...

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

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

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

Studies of Hunter and Hurler syndromes on hold ...

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

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?