“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

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

An experimental gene therapy has become the first treatment to successfully slow the progression of Huntington’s disease. While the findings are still preliminary, the approach could be a major ...

In a trial of people with early Huntington's, the newly developed therapy slowed the progression of symptoms by 75%. reading time 3 minutes Doctors in the UK are claiming to have performed a ...