Breakthrough Gene Therapy Restores Hearing in Deaf Children, Offers Hope for Millions Worldwide

June 6, 2024
Breakthrough Gene Therapy Restores Hearing in Deaf Children, Offers Hope for Millions Worldwide
  • Researchers 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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