Shanghai startup's gene therapy restores hearing in five deaf children, running ahead of Lilly and Regeneron trials
Five of six children who had been deaf since or soon after birth can now hear after receiving a gene therapy developed by Shanghai Refreshgene Therapeutics, according to a new study published in the Lancet Wednesday.
The children in the trial had a mutated version of a gene that encodes otoferlin, disrupting signaling to the brain from the ear’s sound-sensing hair cells. The therapy, which is injected into the inner ear, uses a viral vector known as an AAV to deliver a functional copy of the gene.
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