According to an announcement on January 23rd from Children's Hospital of Philadelphia, 11-year-old patient Aissam Dam heard sounds of life for the first time, such as her father's voice, the sound of passing cars, and the sound of scissors cutting hair.
Aissam Dam was born severely deaf due to an extremely rare genetic defect. Specifically, a faulty gene prevented the production of otoferlin – a protein essential for the hair cells in the inner ear to convert sound vibrations into chemical signals that are transmitted to the brain.
Otoferlin gene defects are very rare, occurring in only 1-8% of cases of congenital hearing loss in children.
Aissam Dam underwent surgery on October 4, 2023. Doctors lifted a portion of her eardrum and injected a harmless virus that had been modified to transport active copies of the Otoferlin gene into the fluid inside her cochlea. As a result, the hair cells began producing the missing protein and functioning normally.
Nearly four months after receiving treatment in one ear, Aissam's hearing has improved significantly. She is now diagnosed with mild to moderate hearing loss and "actually heard sounds for the first time in her life."
According to Surgeon John Germiller, Director of Clinical Research in Otolaryngology at Children's Hospital of Philadelphia, gene therapy for hearing loss is something doctors and scientists have been working towards for over 20 years and have finally succeeded.
"Although the gene therapy we performed on the patient was aimed at correcting an abnormality in a very rare gene, these studies could open up future application opportunities for some of the more than 150 other genes that cause hearing loss in children," said Dr. John Germiller.
Minh Hoa (compiled from Vietnam+, Saigon Giai Phong)
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