Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

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

The trial was small — just 12 children were treated in one or both ears — but it offers important lessons for researchers ...

F or 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 ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

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

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

When she was born, doctors informed Opal Sandy’s parents that their child would never hear. At the age of 11 months, she had ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

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