An experimental gene therapy using harmless viruses has achieved unprecedented progress in treating congenital deafness, paving the way for a new era in the fight against inherited hearing loss.
The largest and longest study of its kind showed that nearly 90% of patients who received the experimental gene therapy regained significant hearing abilities. The trial included 42 patients at eight medical centers in China who had been born with profound hearing loss due to mutations in the OTOF gene. Improvement began within weeks and peaked within a year.
Some patients regained near-normal hearing, while others could hear whispers, and several children even learned to speak for the first time.
The treatment involves delivering a healthy copy of the gene to the inner ear using harmless viruses to restore the function of auditory cells, an approach that differs from cochlear implantation. A two-year follow-up showed that all patients could hear speech, and 60% could hear whispers, with no serious side effects reported.
Researchers emphasize that this achievement represents a turning point, as the treatment can be administered in a single session. Studies are ongoing to expand the scope of the treatment and evaluate its long-term results.