An 11-year-old boy with congenital deafness can now hear sound after he received groundbreaking gene therapy that replaces a mutated gene with the correct version, The New York Times reports.
"There’s no sound I don’t like," said the boy Aissam Dam via interpreters to the NYT. "They’re all good."
Dam's deafness was due to a mutation to a gene called otoferlin, according to the news outlet. The gene makes a protein that's a key component in relaying sound between the inner ear and brain, but a mutated version of the otoferlin gene impedes this process, impacting around 200,000 people across the globe.
In early October last year, Dam was the first person in America to receive the otoferlin gene therapy as part of a clinical study by pharmaceutical company Eli Lilly and Akouos, a gene therapy business Eli Lilly bought in 2022.
At the Children’s Hospital of Philadelphia, researchers injected into one of Dam's ears liquid containing a benign virus carrying functional copies of the otoferlin genes. Specifically, they squirted the normal genes into his cochlea, a spiral-shaped hollow in the inner ear that's filled with liquid and lined with hair cells that send sound information to the brain.
It took a short time — mere days — for the gene therapy to do its miraculous work, the NYT reports. Dam's father said his son was picking up traffic sounds with his treated ear, and now its ability to hear is "close to normal."
Two similar studies were done in China recently, with one of them already showing remarkable results. There are also two studies in Europe that are either in progress or planned that will target the otoferlin mutation.
With Dam's newfound hearing, the NYT reports that researchers are going to continue the study and enroll younger patients.
Even though this particular type of gene therapy targets a rare type of mutation in the inner ear, it also opens up the possibility of treating other forms of congenital deafness.
More on gene therapy for deafness: New Genetic Treatment Successfully Lets Deaf Children Hear
Share This Article