Can gene therapy restore hearing and speech in children born deaf?

This article discusses a post or paper titled "Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results" published by Hui Wang, Yuxin Chen, Jun Lv, Xiaoting Cheng, Qi Cao, Daqi Wang, Longlong Zhang, Biyun Zhu, Min Shen, Chunxin Xu, Mengzhao Xun, Zijing Wang, Honghai Tang, Shaowei Hu, Chong Cui, Luoying Jiang, Yanbo Yin, Luo Guo, Yi Zhou, Lei Han, Ziwen Gao, Jiajia Zhang, Sha Yu, Kaiyu Gao, Jinghan Wang, Bing Chen, Wuqing Wang, Zheng-Yi Chen, Huawei Li & Yilai Shu on the Nature Medicine website on June 5, 2024.

What is the article about?

According to the World Health Organization, over 5% of the global population, or 430 million people, suffer from disabling hearing loss, including 34 million children. There are about 26 million people with congenital hearing loss, of which 60% is attributed to genetic factors. The deficient or dysfunctional otoferlin protein results from pathogenic mutations in the OTOF gene and leads to autosomal recessive deafness 9 (DFNB9).


Why is this information important for you?

This novel gene therapy offers a groundbreaking option for children with inherited deafness, particularly those with mutations in the OTOF gene. By restoring hearing function, the therapy can significantly improve speech perception, sound localization, and overall communication abilities. This advance provides hope for families and healthcare providers seeking effective treatments for congenital hearing loss, emphasizing the importance of early intervention to maximize auditory and developmental benefits.


What are the main take-aways?

  • Efficacy of Bilateral Treatment: Treating both ears with the gene therapy showed additional benefits compared to unilateral treatment, including enhanced speech recognition in noisy environments and the ability to localize sound sources, which are critical for effective communication and daily activities.
  • Promising Results: All five children in the trial experienced significant hearing recovery in both ears, with improvements in speech perception and the ability to appreciate music. These findings highlight the potential of this therapy to dramatically improve the quality of life for children with inherited deafness.
  • Need for Further Research: While the initial results are promising, more extensive trials with larger patient groups and longer follow-up periods are needed to confirm the therapy’s safety and efficacy. The study also underscores the importance of developing robust platforms for testing and refining gene therapies for broader applications.
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