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 ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers.

The neurodegenerative disease damages patients’ motor, cognitive and behavioral controls, which sometimes manifest as ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting ...

After three patients died, the maker of an investigational gene therapy stopped shipments of the drug after it refused to ...

Bayer AG has struck a deal with Atara Biotherapeutics to jointly work on Atara's CAR-T cell anti-tumour treatments, as the German group firms up its commitment to build a specialist cell and gene ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem ...