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Gene Therapy Restores Hearing in Children with Inherited Deafness

Despite significant improvements, questions remain about the longevity of the therapy and its ethical implications.

Overview

  • Experimental gene therapy has successfully restored hearing in several children born with inherited deafness, with studies conducted in both China and the United States.
  • The therapy targets a condition that accounts for 2% to 8% of inherited deafness, caused by mutations in a gene responsible for an inner ear protein called otoferlin.
  • Children treated with the therapy have shown significant improvements in hearing, with one 11-year-old boy able to hear sounds for the first time.
  • Despite the success, questions remain about the longevity of the therapy and whether hearing will continue to improve in the treated children.
  • Some consider gene therapy for deafness ethically problematic, as deafness does not cause severe or deadly illness like other conditions targeted by gene therapy.