BEIJING (dpa-AFX) - In a groundbreaking clinical trial conducted by Mass Eye and Ear in Boston and the Eye & ENT Hospital of Fudan University in Shanghai, gene therapy was successfully implemented in both ears of children aged 1 to 11 years who were born deaf.
This remarkable treatment has demonstrated the potential to fully restore various hearing functions, such as sound localization, distinguishing specific sounds in noisy environments, and developing speech abilities, according to the study published in Nature Medicine.
Prior to this breakthrough, the research team had initiated a trial in December 2022, where they applied gene therapy in a single ear. The recent results emphasize that treating both ears leads to enhanced benefits for children with hereditary deafness known as DFNB9, caused by mutations in the OTOF gene.
This gene failure results in the absence of the otoferlin protein, crucial for transmitting sound signals from the ear to the brain, ultimately leading to hearing and speech impairments.
The gene therapy involved a minimally invasive surgical procedure where doctors administered an injection of the human OTOF gene into the children's inner ears. Following the procedure, the children spent seven to 10 days in the hospital for observation. Within four weeks, they displayed improvements in hearing perception during tests, with gradual progress in speech development.
Although the participants experienced mild adverse effects such as fever and vomiting, they all recovered without any severe complications. The researchers highlighted that this gene therapy is designed as a one-time treatment without the need for repetition, although additional speech therapy may be necessary. The research team is now working towards FDA approval to introduce this gene therapy in the United States and hopes to extend this approach to address other forms of deafness, including noise and age-related hearing impairments.
Copyright(c) 2024 RTTNews.com. All Rights Reserved
Copyright RTT News/dpa-AFX
© 2024 AFX News
