Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time

Aissam Dam, an 11-year-old boy, grew up in a world of profound silence. He was born deaf and had by no means heard something. Whereas residing in a poor neighborhood in Morocco, he expressed himself with an indication language he invented and had no education.

Final yr, after shifting to Spain, his household took him to a listening to specialist, who made a shocking suggestion: Aissam is perhaps eligible for a medical trial utilizing gene remedy.

On Oct. 4, Aissam was handled on the Youngsters’s Hospital of Philadelphia, turning into the primary particular person to get gene remedy in the USA for congenital deafness. The purpose was to supply him with listening to, however the researchers had no thought if the remedy would work or, if it did, how a lot he would hear.

The remedy was a hit, introducing a baby who had identified nothing of sound to a brand new world.

“There’s no sound I don’t like,” Aissam mentioned, with the assistance of interpreters throughout an interview final week. “They’re all good.”

Whereas a whole bunch of hundreds of thousands of individuals on the planet dwell with listening to loss that’s outlined as disabling, Aissam is amongst these whose deafness is congenital. His is an especially uncommon kind, attributable to a mutation in a single gene, otoferlin. Otoferlin deafness impacts about 200,000 folks worldwide.

The purpose of the gene remedy is to switch the mutated otoferlin gene in sufferers’ ears with a useful gene.

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