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 ...

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

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

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

Some genetic disorders—such as cystic fibrosis, hemophilia and Tay Sachs disease—involve many mutations in a person's genome, ...

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

The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the ...

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

NANJING, China — A groundbreaking clinical trial has achieved what many thought impossible: restoring meaningful hearing in people born profoundly deaf, including teenagers and young adults who were ...

New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...

AGC Biologics, your friendly CDMO expert, announced an agreement to provide comprehensive development and manufacturing for Rarity PBC's gene therapy.