An 11-year-old boy experiences hearing for the first time after undergoing breakthrough gene therapy at the Children’s Hospital of Philadelphia.
Born profoundly deaf due to a rare gene abnormality, the treatment involved injecting a modified virus carrying functional copies of the otoferlin gene into his cochlea. This corrected the defective gene and allowed hair cells in the inner ear to produce the essential protein for converting sound vibrations.
Almost four months post-treatment, the boy’s hearing has significantly improved, marking a historic milestone in gene therapy for hearing loss. This holds promise for addressing various genetic causes of childhood hearing loss, offering hope for patients worldwide.