A novel gene therapy has shown promising results in improving hearing for children and adults with a rare genetic form of deafness, known as DFNB9, resulting from OTOF gene mutations. Conducted in China, the multicenter clinical trial enrolled 10 participants aged 1.5 to 23.9 years who received therapy via an engineered virus that delivered a functional copy of the defective gene. Interim results indicate significant hearing improvements were observed within weeks, particularly in children aged 5 to 8, although variations in recovery were noted based on age. The trial included assessments over 6 to 12 months, with most gains occurring in the first month. Ongoing studies will seek to confirm long-term safety and efficacy.

Source: nature medicine