New gene therapy could reduce hearing loss in Norrie disease

child receiving a hearing test A gene therapy developed in mice by an international team of researchers led by UCL and the NIHR Great Ormond Street Hospital Biomedical Research Centre, could significantly reduce hearing loss associated with Norrie disease. Norrie disease is a rare but devastating genetic disorder that causes blindness and hearing loss. The condition primarily affects boys. Children affected by the condition are born blind and develop progressive hearing loss from around the age of 12 years old. The research, published in EMBO , aimed to investigate whether it was possible to slow or prevent this from happening. Currently, the only possible treatment option to help hearing loss in Norrie disease, is cochlear implants. However, these are only partly effective.