6 Deaf Children Can Now Hear After a Single Injection WIRED

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News Source : Wired

News Summary

  • In the future, he says gene therapies might be given in the womb to counteract this early damage.Lustig was an investigator for a gene therapy trial that launched in 2014 that aimed to restore hearing in adults with damaged sensory cells..
  • “We’ve never had a therapy that restores even partial hearing for someone who’s totally deaf other than a cochlear implant.”Video: The Eye & ENT Hospital of Fudan University/The LancetThe children were all born with a mutation in a gene that makes a protein needed for hearing called otoferlin..
  • Eli Lilly announced this week that one of those participants, an 11-year-old boy, could hear within 30 days of receiving an otoferlin gene therapy.And in October, Regeneron’s Decibel Therapeutics in Boston reported improved auditory responses in one patient as part of an ongoing clinical trial..
  • One explanation, Shu says, is that the child had preexisting immunity to the type of virus used to carry the new gene into the inner ear cells—meaning the treatment would have been destroyed by their immune system before it could take effect..
  • Five months later, he spoke his first words.The boy is one of six children with a type of hereditary deafness who are part of a gene therapy trial in China..
  • For some of the parents, it’s the first time they’ve heard their children say “mama” or “baba” (Chinese for “papa”).Other children in the study had previously received a cochlear implant in one ear and had already learned to speak..
To get the new genetic material into cells, they engineered harmless viruses to carry it. Doctors carefully injected a tiny amount of liquid containing the viruses into a part of the childrens inner [+3314 chars]

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