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Deaf girl's parents amazed as gene therapy lets her hear for first time

Deaf girl's parents amazed as gene therapy lets her hear for first time

Summary

An 18-month-old girl named Opal Sandy, born deaf due to a genetic problem, can now hear thanks to a new gene therapy. The treatment involved giving her a healthy copy of the faulty gene, and after several weeks she began responding to quiet sounds and started talking.

Key Facts

  • Opal was born completely deaf because of a mutation in the OTOF gene, which affects hearing.
  • The gene therapy was given in a 16-minute surgery before her first birthday at Cambridge University.
  • The therapy delivered a working copy of the OTOF gene directly to her right ear.
  • Within weeks, Opal could hear loud sounds; after 24 weeks, she could hear whispers and near-normal sounds.
  • After treatment, Opal stopped using her cochlear implant, a device that helps deaf people hear.
  • Opal started saying simple words like “Mama” and “Dada” following the therapy.
  • The gene therapy is part of a trial involving children in the UK, U.S., and Spain.
  • The therapy is produced by American company Regeneron and has shown no adverse effects in Opal.
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