Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
Nature • • general
Key Points:
- Neutralizing antibody titers against AAV1 were tracked in participants over 2.5 years, showing overall trends and individual variations with data presented as median ± IQR.
- Hearing thresholds measured by ABR, ASSR, and behavioral audiometry improved over time in participants with hearing recovery, with statistical analysis confirming significant changes from baseline up to 2.5 years.
- Comparison of two dose groups (1.5 × 10¹² and 2.1 × 10¹² vg/ear) showed hearing threshold improvements in both, though the higher dose group had shorter follow-up due to later treatment initiation.
- Age-related analysis revealed differences in hearing threshold improvements across four age groups, with mean changes calculated from individual baselines for ABR, ASSR, and behavioral audiometry.
- Baseline distortion product otoacoustic emission (DPOAE) signal-to-noise ratio correlated with improved ABR and ASSR thresholds at 26 weeks, indicating baseline cochlear function may predict hearing recovery outcomes.
