"Breakthrough Gene Therapy Restores Hearing in Children with Genetic Deafness"

1 min read
Source: NPR
"Breakthrough Gene Therapy Restores Hearing in Children with Genetic Deafness"
Photo: NPR
TL;DR Summary

An experimental gene therapy has shown promise in restoring some hearing for children with an inherited form of deafness caused by a rare genetic defect in the otoferlin gene. The therapy, which involves modifying a virus to carry a functioning form of the gene into the inner ear, resulted in improved hearing for five out of six children involved in the study. While the children's hearing isn't completely normal, the treatment appears safe and the researchers are hopeful that the improvement is permanent, marking a significant advancement in the treatment of hereditary deafness.

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