His father's voice, the roar of cars, the sound of scissors cutting his hair: an 11-year-old boy is hearing “for the first time in his life” after a new gene therapy, a Philadelphia hospital said Tuesday. UNITED STATES .
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This achievement, a first in the country, represents hope for patients around the world suffering from hearing loss caused by genetic mutations, Children's Hospital of Philadelphia (CHOP), which provided the treatment, said in a news release.
Aissam Dam was born “profoundly deaf” due to a very rare anomaly in a single gene.
“Gene therapy for hearing loss is a goal that we as hearing loss doctors and scientists have been pursuing for more than 20 years. And we finally did it,” said surgeon John Germiller, head of clinical research in the Department of Otorhinolaryngology (ENT).
“The gene therapy we used on our patient was aimed at correcting the abnormality of a very rare gene, but these studies could pave the way for the future use of more than 150 other genes responsible for hearing loss in children “, he said happily in the press release.
In patients like Aissam Dam, a defective gene prevents the production of otoferlin, a protein that hair cells in the inner ear need to convert sound vibrations into chemical signals that are sent to the brain.
Abnormalities of the otoferlin gene are very rare, accounting for between 1 and 8% of cases of hearing loss at birth.
On October 4, 2023, the boy underwent surgery to partially lift his eardrum and then inject a harmless virus into the internal fluid of his cochlea that had been modified to carry working copies of the otoferlin gene.
The hair cells then began to produce the missing protein.
Almost four months later, Aissam's hearing has improved to the point where he only has mild to moderate hearing loss.
Aissam was born in Morocco before moving to Spain with his family. However, he may never be able to speak because the part of the brain responsible for language acquisition shuts down around the age of five, reports The New York Times.
Other similar studies in children are underway or about to begin in the United States, Europe and China, some of which have been successful.