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How gene therapy turned on a boy's hearing

A mutant gene has been replaced by a normal one to enable a child deaf from birth to hear.

A new gene therapy treatment has allowed an 11-year-old boy from Morocco, who was born deaf due to a mutant gene, to - in effect - turn his hearing on.

As part of an international clinical trial, doctors at the Children’s Hospital in Philadelphia delivered a single, small dose of gene therapy containing copies of the normal gene directly to the inner ear of Aissam Dam.

Now, almost four months after receiving the investigational gene therapy in one ear, Aissam – who was born profoundly deaf – has only mild to moderate hearing loss in the treated ear.

Dr John Germiller, one of the doctors at the Children's Hospital of Philadelphia who carried out the procedure, told Newsday about Aissam and the potential for gene therapy to cure other patients with some form of genetic hearing loss.

“One thing that we know about children like him is if they don't get sound information early in life, they won't learn to speak a spoken language. He'll never develop that... but he'll hear everything that we hear, but if you speak to him he'll never be able to understand language. But he will know someone is speaking to him, which is revolutionary for him. It was remarkable how well he improved. It proves the concept and opens that door."

(Pic: Aissam Dam; Credit: Children’s Hospital of Philadelphia)

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