- Gene therapy developed to treat autosomal recessive deafness type 9.
- Utilizes a synthetic adeno-associated virus for gene delivery.
- 10 participants aged 1.5 to 23.9 years enrolled.
- Significant hearing improvements observed, especially in ages 5-8.
- Most gains occurred within the first month post-treatment.
- Safety profile showed mostly mild to moderate adverse events.
- Ongoing assessments for long-term effects and durability planned.
Source: nature medicine