June 5th 2024.
Ground-breaking gene therapy has successfully restored hearing in two deaf children in China, allowing them to listen to music and even dance to it. This pioneering treatment, which involves replacing faulty DNA in the ear, has been given to a small group of patients, making them the first in the world to receive it.
The therapy has been administered as an infusion into the ear, targeting the specific type of inherited deafness called DFNB9. Out of the five children who received the treatment, two girls and three boys were able to hear and speak words, as well as locate the source of sound. Remarkably, two of them even showed significant progress in responding to music, which has more complex sound signals, according to the researchers.
Zheng-Yi Chen, an associate scientist at Mass Eye and Ear in the US, expressed amazement at the results, stating that the treated children's hearing abilities continue to improve. The team also noted that administering the therapy in both ears has additional benefits, such as giving the children the ability to hear sound in three dimensions, which is crucial for communication and everyday tasks like driving.
The study, published in the journal Nature Medicine, has shown great promise and has prompted the researchers to call for larger international trials. While this gene therapy currently only targets DFNB9, there is hope that similar treatments can be developed for other types of deafness caused by genetic factors.
This latest research builds upon the team's previous work, where they successfully treated children in one ear. After receiving the gene therapy, these children went from being profoundly deaf to having mild to moderate hearing loss in the treated ear. However, administering the therapy in both ears resulted in even more benefits, including the ability to hear in noisy environments.
DFNB9 is one of the most common forms of hereditary deafness, affecting an estimated 20,000 people in Europe and the US. It is caused by a lack of a protein called otoferlin, which is crucial for transmitting sound signals from the ear to the brain. Currently, the only recommended treatment for DFNB9 is a cochlear implant.
To deliver the gene therapy, the researchers used a modified version of the adeno-associated virus, which is harmless to humans. They modified it to carry a version of the human OTOF gene, which was then infused into the inner ears of the patients. The children were monitored for 13 or 26 weeks at the Eye & ENT Hospital of Fudan University in Shanghai, China, and no major side effects were reported. The trial is still ongoing, with the patients being closely monitored for further progress.
The team acknowledges that more work needs to be done to refine this therapy, but they are encouraged by the results so far. In a separate clinical trial at Addenbrooke's Hospital in Cambridge, a girl in the UK also received a similar gene therapy, and she has shown significant improvement in her hearing abilities. Opal Sandy, 18 months, who received the treatment in one ear and a cochlear implant in the other, can now hear loud sounds and is starting to say words like "mama" and "dada." This groundbreaking research offers hope for the future of treating hereditary deafness and improving the lives of many more children.
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