An 11-year-old boy, Aissam Dam, has regained his hearing after receiving a groundbreaking gene therapy at the Children’s Hospital of Philadelphia (CHOP).
The therapy, a first in the United States, corrected a rare gene abnormality that caused his profound deafness.
The treatment involved a surgical procedure to inject a modified virus carrying a working copy of the otoferlin gene into his cochlea, enabling the hair cells to produce the missing protein.
“Gene therapy for hearing loss is something that we physicians and scientists in the world of hearing loss have been working toward for over 20 years, and it is finally here,” surgeon John Germiller said.
“While the gene therapy we performed in our patient was to correct an abnormality in one, very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss.”
“As more patients at different ages are treated with this gene therapy, researchers will learn more about the degree to which hearing is improved and whether that level of hearing can be sustained over many years,” Germiller said.
Aissam’s hearing has improved significantly, but due to his age, he may not learn to talk.
The study, sponsored by Akouos, Inc, is part of ongoing research to understand the long-term effects of this gene therapy on hearing improvement.
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