New gene therapy restores natural hearing for deafness at birth
Nov. 06, 2023.
1 min. read Interactions
... but only works for patients with otoferln deficiency and may cost up to $250,000
Several Chinese children with deafness at birth have had their natural hearing pathway restored with new gene therapy, MIT Technology Review reported last week.
“Shu Yilai, a surgeon and scientist at Fudan University in Shanghai, who is leading the experiment, explains that the new treatment applies only to one specific cause of deafness at birth: a defect in a gene that produces a protein called otoferlin. … “In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears.”
Hair cells + otoferlin relay sounds to the brain
“The inner ear contains about 16,000 hair cells, vibrating at different frequencies of sound. Without otoferlin, these cells cannot transmit the chemicals that relay information to the brain,” MIT Technology Review explains.
“The new treatment is designed to add a working copy of the otoferlin gene. Yilai injects the loaded viruses deep into a fluid-filled chamber in a part of the ears called the cochlea. … The treatment reaches maybe 60% to 65% of normal hearing,’ says Yilai.”
Today (Monday, November 6), MIT Technology Review clarified that “gene therapy remains relatively expensive and inaccessible. … Nova Liu, the founder of Shanghai Refreshgene Therapeutics, which financially backed the Chinese research on this gene therapy, estimated that a commercialized product could cost between $125,000 and $250,000.”
Also, “this particular gene therapy works only when the deafness is due to a lack of otoferlin. … Only around 1% to 5% of cases of inborn deafness are caused by lack of otoferlin, so many more deaf children aren’t able to benefit from this therapy yet.”