Summary of Gene Therapy Study for Restoration of Hearing to Babies Born Deaf
This study was published by Nature Medicine on June 5th, 2024. The story presents evidence that gene therapy was successful in terms of restoring hearing in babies that were born deaf due to DFNB9. It is said that this is linked to a genetic condition to mutations in the gene OTOF. The study was lead and conducted by Dr. Yilai Shu, from Eye & ENT hospital of Fudan University in Shanghai. This study advanced the treatment of inherited deafness tenfold, especially due to the fact that there are currently zero options provided by pharmacies to treat this.
Patients
The patients involved in this study included five babies, all with DFNB9. All patients received gene therapy to restore hearing in both ears simultaneously. It is said that DFNB9 comes from the inability to produce working otoferlin protein that is needed for auditory and nerve function related to hearing.
Gene Therapy Method
The children were treated with injections of an altered virus that carried healthy copies of the OTOF gene. The injections were placed directly into the inner ears of the children. The tactic behind this is approach is that the functional gene copies were supposed to replace the defective ones. Thus, allowing for proper hearing.
Results
All five of the children showed tremendous improvement in their hearing abilities. This in turn helped with speech and localizing sound. Two of the children were found to have a new appreciation for music which led to dancing.
Safety and Significance
The children were monitored for 13 to 26 weeks during this study. After the study was concluded, it is important to state that no adverse effects or harm was caused to these children. The study allows for the potential of gene therapy to change the lives of those with genetically impaired hearing.
Conclusion
In conclusion, the study published in Nature Medicine represents a pivotal advancement in medical research, offering hope to millions affected by genetic deafness worldwide. The success of restoring hearing in children born deaf through gene therapy not only validates the approach but also paves the way for further research and clinical trials aimed at enhancing auditory capabilities across diverse populations. As researchers continue to refine and expand this treatment modality.
Review Article Support
I believe that the review article “Understanding and treating pediatric hearing impairment” supports the information the magazine article. Both articles mention that at least 50% of hearing impairment is genetic. The review article discusses monogenic deafness as a future possibility for gene therapy. Gene therapy relates to the magazine topic of the OTOF gene.
The review article intertwines with the magazine article because it supports the idea of gene therapy.
Citations
Thompson, D. ( 2024, June 5th) Gene Therapy in Both Ears Restores Hearing to Babies Born Deaf: U.S.News & World Report
Wrobel, Christian. Et el. Understanding and treating pediatric hearing impairment. EBioMedicine. DOI: 10.1016/j.ebiom.2020.103171