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

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 ...

Up to 60% of congenital and early-onset hearing loss is caused by genetic mutations in an inherited gene, and gene therapy ...

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

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

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

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

The global cell and gene therapy market is undergoing rapid expansion, driven by the surge in regulatory approvals and continual advancements in therapeutic technologies. The market is anticipated to ...

Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same ...

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

A one-time subretinal injection of Sura-vec at both high- and low-dose concentrations allowed more than half of patients with ...