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Thanks to gene therapy, they restored the hearing of an 11-year-old boy. “This is the first case that we have managed to cure”

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He has been deaf since birth, but now – thanks to American doctors – he is learning about the world of sounds. Specialists from the Children’s Hospital of Philadelphia used experimental gene therapy, thanks to which an 11-year-old boy can hear. At first, Aissam was terrified and overwhelmed, but he admits that there is no sound he doesn’t like. What he likes most is listening to people.

Aissam grew up in a world without sounds. He grew up in a small Moroccan village. Until the age of eight, he did not go to school and communicated using sign language, which he invented. Thanks to experimental gene therapy and doctors at the Children’s Hospital of Philadelphia, 11-year-old Aissam heard his father’s voice for the first time. – He hears people’s voices and approaching cars. He is very pleased. It’s new to him, so he was a little scared at first, says Dr. John A. Germiller of the Children’s Hospital of Philadelphia.

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The boy was born with congenital deafness caused by a mutation in the otoferlin gene, which is needed by the hair cells of the inner ear to transmit sounds to the brain. The aim of the therapy was to replace the defective gene with a working copy.

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To restore the 11-year-old’s hearing, surgeons injected him with a harmless virus into his ear that had been modified to transport working copies of the gene responsible for the production of otoferlin. This allowed the hair cells to produce the missing protein and function properly. – It is estimated that there are over 150 genes that cause hearing loss at birth. This is the first case that we have managed to cure – admits Dr. John A. Germiller.

A great success for Polish doctors. Their reconstructive surgery was rewardedMarek Nowicki/Fakty TVN

Unprecedented success

In many other deafness-causing mutations, hair cells die in infants and even in utero. However, in the case of deafness caused by a mutation in the otoferlin gene, these cells can survive for years, allowing time for gene therapy to be used.

Aissam, who had never had a cochlear implant, began hearing his first sounds just a few days after the procedure. “There is no sound I don’t like. They’re all great,” The New York Times quotes the boy.

Doctors openly admit that the boy may never speak, because learning to speak occurs in small children. But thanks to gene therapy, his quality of life will significantly improve. – His father is happy that Aissam will know when someone is talking to him. Even if he doesn’t understand the words, he will be aware, for example, that someone is standing behind him. There are also safety issues. The boy will hear cars passing by, emphasizes Dr. John A. Germiller.

READ ALSO: Groundbreaking surgery in New York. “It’s something doctors have thought about for centuries but have never done before.”

34 million children around the world cannot hear at all or have hearing loss. Genes are responsible for 60 percent of cases. However, defects in the otoferlin gene are rare. Due to this mutation, approximately 200,000 people around the world are deaf. The success of Philadelphia doctors is a milestone that gives hope to these patients. “We’ve been waiting for this for a long time. For decades, people have been asking when it would work. I didn’t think gene therapy would start in my lifetime, but it did,” says Dr. Margaret A. Kenna of Children’s Hospital to The New York Times in Boston.

Similar research is being conducted in Asia and Europe. On Wednesday, The Lancet published a study showing that gene therapy helped restore hearing to five children in China. More patients are waiting for treatment at a hospital in Philadelphia – a 3-year-old from Florida and a 3-year-old girl from California.

Author:Justyna Kazimierczak

Facts about the World TVN24 BiS

Main photo source: Reuters



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