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Gene Therapy Restores Hearing in Children with Hereditary Deafness

Innovative treatment shows promise in reversing hearing loss caused by OTOF gene mutations, offering new hope for affected families.

  • Five children regained hearing after receiving AAV-based gene therapy in both ears.
  • The therapy targets mutations in the OTOF gene, essential for transmitting sound signals.
  • Children showed improved speech perception and ability to locate sounds in noisy environments.
  • The trial, conducted in Shanghai and Boston, marks the first bilateral application of this therapy.
  • Researchers aim to expand trials and seek FDA approval for broader use.
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