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  • 1440 Daily Digest

Hearing Loss Breakthrough



An 11-year-old boy with congenital hearing loss is able to hear for the first time thanks to an experimental gene therapy treatment, according to initial results released yesterday.


Aissam Dam was born with hearing loss caused by a mutated single gene known as otoferlin, which affects roughly 200,000 people globally. Otoferlin is a protein in the inner ear's hair cells that allows for sound to be transmitted to the brain. Dam became the first patient in the US to undergo a surgical procedure at the Children's Hospital of Philadelphia in October, in which doctors replaced the mutated otoferlin gene in Dam's ears with a functional gene. The gene therapy trial showed Dam's hearing was restored within 30 days of the procedure, reducing his hearing loss severity from total to mild (see chart).


The trial from Philadelphia is one of five that are underway, with others in China and Europe. Results from all five trials are set to be presented next month. See the science behind hearing here (via YouTube).

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