A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions.

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington’s disease. The news has spread quickly through families affected by this condition, who have ...

Share on Pinterest The new gene therapy showed promise by significantly slowing down Huntington’s disease. Santi Nuñez/Stocksy Huntington’s disease is a genetic brain disorder that causes symptoms ...

An experimental gene therapy was found to slow the progression of Huntington's disease, marking a major step toward a potential first genetic treatment for the condition.

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased ...

The first participant in an investigational gene therapy trial of STXBP1 encephalopathy died, Capsida Biotherapeutics said. The death occurred days after dosing, according to information posted on the ...

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

Life sciences ranked among the top four sectors, with a notable rise of companies integrating AI and biotech in transformative ways. One example is Insilico Medicine, a global AI-driven company that ...

A technique to estimate the health of specialized nerve cells may help predict patient response to Zolgensma gene therapy, a study reports.

Love is in the air, but Jasper has a case to solve when death strikes within the university. In the lead-up to Zelda’s wedding, Jasper is wrestling with his feelings for Helena. Dan becomes obsessed ...