Breakthrough Gene Therapy Restores Hearing in Deaf Children, Offers Hope for Millions Worldwide
June 6, 2024Researchers from Mass Eye and Ear and Fudan University have restored hearing in five children through a gene therapy trial targeting mutations in the otoferlin gene.
The therapy involved providing a normal copy of the mutated gene, improving hearing levels from 50 to 60 percent of normal.
This dual AAV approach has shown promising results in reversing deafness caused by DFNB9, with similar trials underway in the UK and the US.
The treatment has minimal side effects and potential to help millions worldwide with hearing loss.
Researchers are now focusing on long-term follow-up studies and seeking FDA approval to bring the therapy to the U.S.
The National Deaf Children's Society emphasizes the importance of informed decision-making for families considering gene therapy for deafness.
Summary based on 7 sources
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Sources
The Guardian • Jun 5, 2024
Gene therapy trial restores hearing in both ears for deaf childrenNew Scientist • Jun 5, 2024
Gene therapy enables five children who were born deaf to hearMedical Xpress • Jun 5, 2024
Gene therapy trial restores hearing in both ears of children who were born deaf