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

Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

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.

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Researchers in the United Kingdom say they have successfully trialed what could become the world's first gene therapy for Huntington's disease – a fatal neurodegenerative disorder that is typically ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...

Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for ...

The Integrated Health Clinic at University Medical Center New Orleans with LSU Health New Orleans is one of only six teams ...

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends. It was a contrast to how Cole, 18, spent part of the 2024 holiday ...