Being able to reduce hearing loss would be a great achievement, and this would be able to make a difference in the lives of a lot of people, especially children. Here are the latest reports about a true miracle – gene therapy.
Gene therapy and hearing loss
A team of international researchers, led by UCL and the NIHR Great Ormond Street Hospital Biomedical Research Centre, has successfully created a gene therapy for mice that significantly reduces the hearing loss associated with Norrie disease.
Norrie disease is a rare genetic disorder that causes blindness and hearing loss, predominantly affecting boys. Children with Norrie disease are born blind and develop progressive hearing loss from the age of 12.
This research aims to slow down or prevent hearing loss, which can cause dual sensory deprivation and severely restrict the lives of those with Norrie disease.
At present, cochlear implants are the only available treatment for Norrie disease, but their effectiveness is limited. Researchers have conducted a study on mice, which was published in EMBO, demonstrating the use of gene therapy to prevent the death of hair cells in the cochlea. The cochlea is responsible for hearing and shaped like a snail shell.
This therapy can also stop hearing loss associated with Norrie disease. The team administered a gene therapy that can restore the missing norrin protein.
The treatment was shown to improve the development of abnormal blood vessels in the ears and eyes of mice with Norrie disease. This gene therapy could help prevent deafblindness as hearing and vision tests conducted on mice showed improvement after treatment.
In individuals with Norrie disease, a faulty gene called NDP causes a gradual loss of hearing due to its impact on certain structures within the inner ear.
Through our research with mice who have Norrie disease, we have discovered a potential solution by introducing billions of corrected copies of the gene into the bloodstream. This gene therapy has shown promising results in improving the inner ear’s blood vessel structure and protecting the sensory hairs responsible for hearing.
Dr. James Wawrzynski, a co-author of the study from UCL Great Ormond Street Institute of Child Health, highlights the importance of providing effective treatment for children with visual impairments.
This can potentially change their lives and benefit those in hospitals worldwide, including GOSH. The challenges of being blind can be alleviated by slowing or treating its progression.
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