Many infants who were born deaf can now hear, thanks to gene therapy. Five out of the six children treated in China had their hearing largely restored, according to a tiny study released on Wednesday.
Analogously, an 11-year-old kid receiving treatment at the Children’s Hospital of Philadelphia reported comparable progress on Tuesday. Additionally, Chinese researchers released a study earlier this month that revealed similar findings in two additional kids.Â
The experimental treatments currently only address one uncommon ailment. However, researchers believe comparable therapies may one day benefit many children with different genetically based forms of deafness. 34 million children worldwide suffer from deafness or hearing loss, with up to 60% of instances being attributed to genetics.
Gene therapy, which has already been approved to treat conditions including sickle cell disease and severe hemophilia, is now being used to treat the latest problem that scientists are targeting hereditary deafness.Â
Kids who are born deaf frequently receive a cochlear implant, a technology that lets them hear sounds. “Hearing loss cannot be reversed by medication… Zheng-Yi Chen of Boston’s Mass Eye and Ear, a senior author of the study published on Wednesday in the journal Lancet, stated, “That’s why we were always trying to develop a therapy.” “We couldn’t be more happy or excited about the results.”Â
The group made footage of the patients’ advancement. Six weeks following therapy, one depicts a newborn who was previously completely deaf, looking back in response to a doctor’s remarks. Another depicts a young child who, thirteen weeks into her treatment, keeps repeating her father, mother, sister, grandma, and “I love you.”Â
An 11-year-old child who was born deaf at birth and ready for gene therapy surgery in Philadelphia in October 2023. The hospital declared on Tuesday, January 23, 2024, that he is now experiencing sound for the first time in his life and that his hearing loss in the treated ear is now limited to mild to moderate. Â
Every child involved in the studies suffers from a syndrome that explains between 2 and 8% of hereditary deafness. Mutations in this gene lead to the production of otoferlin, an inner ear protein that aids in the transmission of sound waves from hair cells to the brain. During a surgical operation, a functional copy of that gene is delivered to the inner ear via the one-time therapy. While one child in the two-person research received treatment in both ears, most of the children received treatment in one ear.Â
The six-child study was conducted at Fudan University in Shanghai, and it was co-led by Dr. Yilai Shu, a former student of Chen’s group. Funders include Shanghai Refresh gene Therapeutics, a biotech firm, and Chinese science groups.Â
The kids were under the researchers’ care for roughly six months. They are unsure of the reason behind one of them’s treatment’s failure. However, the five others, who were completely deaf before, are now able to hear and interact with others during a normal conversation. Chen estimates their current level of hearing to be between 60% and 70% of normal. There were no serious negative effects from the therapy.Â
Journal Reference Â
- Qi, F. Tan, L. Zhang, L. Lu, S. Zhang, Y. Zhai, Y. Lu, X. Qian, W. Dong, Y. Zhou, Z. Zhang, X. Yang, L. Jiang, C. Yu, J. Liu, T. Chen, L. Wu, C. Tan, S. Sun, H. Song, Y. Shu, L. Xu, X. Gao, H. Li, R. Chai, AAV-Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness. Adv. Sci. 2024, 2306788. https://doi.org/10.1002/advs.202306788.Â
