Today's scientific advancements are paving the way for groundbreaking innovation and challenging the status quo. Recent strides in gene therapy, particularly with the otoferlin gene, have sparked questions for parents of children with hearing loss and the professionals who work with them. Because this information on genetic therapies is so new, there are more questions than answers on what these scientific breakthroughs mean for children with hearing loss today.
Hearing First, an initiative of the Oberkotter Foundation that provides education and support to families and professionals, released a family-friendly video and article to explain the study and explore what it means for the future of childhood hearing loss.
Click here to read the article and watch the video.
About the Recent Gene Therapy Studies
In early 2024, two medical journal articles talked about two different gene therapy studies conducted in China1 2. The studies included children with a unique type of deafness caused by a mutation in a gene called the otoferlin gene.
Many of the children in these studies had a considerable improvement in their hearing after receiving genetic therapy. In some cases, these children went from being completely deaf to being able to hear in the mild hearing loss range. Also, some of these children showed improvement in their ability to respond to speech. A report in The New York Times talked about a similar otoferlin genetic therapy study happening in the United States, under the watch of the FDA.
As we find ourselves at the forefront of future pediatric hearing healthcare and navigating this new frontier of research, one thing is certain: the conversation surrounding gene therapies and their role in addressing childhood hearing loss is just beginning.
Those interested in learning more about the role of genetics in hearing loss can take a course from Hearing First, Genetics and Hearing Loss: Fundamentals of Genetics, at no cost.
- Lv, J., Wang, S., Cheng, X., Chen, Y., Wang, D., Zhang, L., Cao, Q., Tang, H., Hu, S., Gao, K., Xun, M., Wang, J., Wang, Z., Zhu, B., Cui, C., Gao, Z., Guo, L., Yu, S., Jiang, L., . . . Shu, Y. (2024). AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial. Lancet. https://doi.org/10.1016/s0140-6736(23)02874-x
- Qi, J., Tan, F., Zhang, L., Lü, L., Zhang, S., Zhai, Y. C., Lu, Y., Qian, X., Dong, W., Zhou, Y., Zhang, Z., Yang, X., Jiang, L., Yu, C., Liu, J., Tian, C., Wu, L., Tan, C., Sun, S., . . . Chai, R. (2024). AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness. Advanced Science, 11(11). https://doi.org/10.1002/advs.202306788