Duke Eye Center has become the first academic medical center in the country to implant a new cell-based gene therapy for a rare eye disease called MacTel. This procedure offers hope to patients like ...

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

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

ZEVASKYN ® (prademagene zamikeracel) is an autologous cell sheet-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis ...

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

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

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

Data demonstrates sustained clinical improvements in pain, stiffness and function for up to 156 weeks following a single ...

These results are what we hoped for when we first began developing this approach,' one researcher said.View on euronews ...

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

On her mother’s instruction, a toddler gives her pink stuffed horse, Snap Snap, a big cuddle. Then, as told, she plants a kiss on his soft fur and, because he’s hungry, feeds him a pretend bowl of ...