Chinese and US scientists achieved near-simultaneous breakthroughs in using gene therapy to treat deafness

~200,000 people suffer globally from a rare, congenital (from birth) genetic disorder that causes a mutation in a single gene, otoferlin

That disrupts communication between the ear and the brain, resulting in complete deafness

For decades, scientists have experimented with gene therapy, or transplanting normal genes into cells to replace missing or defective ones

Until recently, though, researchers had never developed gene therapies for hearing loss, considering the ear to be a particularly complex and challenging body part

Now, researchers in the US, China, and Europe are developing gene therapies to treat otoferlin deafness, which is considered relatively simple to treat because it’s linked to a single gene

Earlier this week, researchers at the Children’s Hospital of Philadelphia reported that late last year, they used a novel gene therapy to restore hearing in an 11-year-old boy

The boy, Aissam, was born with otoferlin deafness

Since the October 4, 2023 operation, though – the first in the US to use gene therapy to treat congenital deafness – Aissam has developed the ability to hear

“There’s no sound I don’t like. They’re all good,” Aissam told the New York Times in an interview

Then on Wednesday, a team of Chinese and US researchers announced it had achieved a similar breakthrough

In a study published in The Lancet, an academic journal, they said that using gene therapy, they improved hearing in five of six children born with otoferlin deafness

Four of the children – aged one to seven – reported “robust” improvements, while all five who responded to the gene therapy treatment also showed improvements in speech perception

Researchers from both teams expressed optimism the results could usher in a new era of gene therapies to treat deafness

Researchers from the US, China, and Europe are expected to share the results of their respective gene therapy trials to treat deafness at an academic conference in February

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