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 from both ears he was able to hear his father's voice for the first time thanks to the effects of gene therapy applied to hearing loss. The little one was cared for Children's Hospital of Philadelphia (Chop), who reported it.
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 (accounts for 1-8% of hearing loss present at 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 so that the hair cells of the inner ear can 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. Consequentially, the hair cells began producing the missing protein and to function correctly. It has been 4 months since the surgery and the patient's current hearing loss has already improved enough to be classified as only mild to moderate, when it was total.
Tested on older children
There Food and Drug Administration American, which gave the green light to the study, wanted to start the research on the first older childrenfor safety reasons, although the window for acquiring speech closes around the age of five. The 11-year-old may not learn to speakthen, but now hears sounds: As more and more patients of different ages are treated with this gene therapy, researchers will learn more about the degree of hearing improvement and whether that level of hearing can be sustained for many years, Germiller said.
Corriere della Sera also on Whatsapp. sufficient click here to subscribe to the channel and always be updated.
January 25, 2024 (modified January 25, 2024 | 3:22 pm)
© ALL RIGHTS RESERVED
#Gene #therapy #11yearold #born #deaf #hears #father39s #voice #time
