by Health Editorial Staff
The defective gene that prevented the child from producing the otoferlin protein, essential for hearing sounds, was replaced with the viral vector technique. Prospects for the treatment of the other 150 genes that cause deafness
An 11-year-old boy born deaf in both ears has managed to hear his father’s voice for the first time thanks to the effects of gene therapy applied to hearing loss. The little boy was treated at the Children’s Hospital of Philadelphia (Chop), which announced the news.
The prospects
a therapy we have been working on for over 20 years and is finally here, said surgeon John Germiller, director of clinical research for CHOP’s otolaryngology division. While the gene therapy we performed on our patient aimed to correct an abnormality in a very rare gene (representing the cause of 1-8% of hearing loss present from birth), these studies could open the door to future use for some of the more than 150 other genes that cause childhood hearing loss.
The defective gene replaced
The child born deaf due to a defective gene prevents the production of otoferlin, a protein necessary for the hair cells of the inner ear to convert sound vibrations into chemical signals that are sent to the brain. Gene therapy replaced the faulty gene within the cell’s DNA with the healthy gene, which was inserted into the ear via a harmless virus, used as a vector and modified to carry working copies of the otoferlin gene. As a result, the hair cells began to produce the missing protein and function properly. 4 months have passed since the surgery and the patient’s current hearing loss has already improved so much that it was only classified as mild to moderate, when it was total.
Tested on older children
The US Food and Drug Administration, which gave the green light to the study, wanted to start the research on older children first, for safety reasons, even though the window for acquiring speech closes around five years of age. The 11-year-old may not learn to speak, then, but now he hears sounds: As more patients of different ages are treated with this gene therapy, researchers will learn more about how much hearing improves and whether that level of hearing improves. hearing can be sustained for many years, Germiller said.
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January 25, 2024 (modified January 25, 2024 | 3:22 pm)
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