His father’s voice, the roar of cars, the sound of scissors cutting his hair: an 11-year-old boy hears “for the first time in his life” after unprecedented gene therapy, a hospital in Philadelphia, United States.
This achievement, the first in the country, represents hope for patients around the world who suffer from hearing loss caused by genetic mutations, the Children’s Hospital of Philadelphia (CHOP), which carried out the treatment, said in a statement.
Gene therapy
Aissam Dam was born “profoundly deaf” due to a very rare abnormality in a single gene. Abnormalities of the otoferlin gene are very rare and account for between 1 and 8% of cases of hearing loss at birth.
“Gene therapy for hearing loss is a goal that we, as hearing loss physicians and scientists, have been pursuing for more than 20 years. And we have finally achieved it,” said surgeon John Germiller, director of clinical research at otorhinolaryngology (ENT). division.
“The gene therapy we applied to 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 genes responsible for hearing loss in children,” he rejoices. the press release. .
In patients like Aissam Dam, a defective gene prevents the production of otoferlin, a protein needed by hair cells in the inner ear to convert sound vibrations into chemical signals sent to the brain.
On October 4, 2023, the boy underwent surgery that partially lifted his eardrum and then injected the internal fluid of his cochlea with a harmless virus that had been modified to carry functional 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 that she now only has mild to moderate hearing loss.
Born in Morocco before moving to Spain with his family, Aissam may never be able to speak, as the part of the brain intended for speech acquisition shuts down around age five, the New York Times reports.
Other similar studies with children are underway or about to begin in the United States, Europe, and China, some of which have been successful.
Source: BFM TV
