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Breakthrough gene therapy allows 11-year-old boy to hear for the first time – Times of India

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Aissam Dam, an 11-year-old boy, grew up in a world of profound silence. He was born deaf and had never heard anything. While living in a poor community in Morocco, he expressed himself with a sign language he invented and had no schooling. Last year, after moving to Spain, his family took him to a hearing specialist, who made a surprising suggestion: Aissam may be eligible for a clinical trial using gene therapy.
On Oct 4, Aissam was treated at the Children’s Hospital of Philadelphia, becoming the first person to get gene therapy in the US for congenital deafness. The goal was to provide him with hearing, but the researchers had no idea if the treatment would work or, if it did, how much he would hear. The treatment was a success, introducing a child who had known nothing of sound to a new world. “There’s no sound I don’t like,” Aissam said, with the help of interpreters during an interview last week. “They’re all good.”
While hundreds of millions of people in the world live with hearing loss that is defined as disabling, Aissam is among those whose deafness is congenital. His is an extremely rare form, caused by a mutation in a single gene, otoferlin. Otoferlin deafness affects about 2,00,000 people worldwide. The goal of the gene therapy is to replace the mutated otoferlin gene in patients’ ears with a functional gene. Although it will take years for doctors to sign up many more patients – and younger ones – to further test the therapy, researchers said that success for patients like Aissam could lead to gene therapies that target other forms of congenital deafness. It is a “groundbreaking” study, said Dr. Dylan Chan, a pediatric otolaryngologist not involved in the trial.
The one in which Aissam participated is supported by Eli Lilly and a small biotechnology firm it owns, Akouos. Aissam’s trial is one of five that are either underway (others are in China and Europe) or about to start.
In patients like Aissam, a defective gene prevents the production of otoferlin, a protein necessary for the “hair cells” of the inner ear to be able to convert sound vibrations into chemical signals that are sent to the brain. The gene therapy consists of a harmless virus carrying new otoferlin genes in two drops of liquid that are delicately injected down the length of the cochlea, delivering the genes to each hair cell. Despite the success, the researchers recognise that Aissam may never be able to understand or speak a language, said Dr. John Germiller, an otolaryngologist who led the study. The brain’s window for acquiring speech closes around the age of five, he said. But when asked if there was a sound he particularly liked, Aissam did not hesitate. “People,” he said in sign language.





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