Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
Nature • • business
Key Points:
- Neutralizing antibody titers against AAV1 were tracked in participants over 2.5 years, showing overall trends and individual trajectories 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 significance; data were collected up to 2.5 years post-treatment.
- Comparison between two AAV dose groups (1.5 × 10¹² and 2.1 × 10¹² vg/ear) showed hearing recovery outcomes with shorter follow-up in the higher dose group due to later treatment initiation.
- Hearing threshold improvements varied across age groups, with mean changes from baseline analyzed for ABR, ASSR, and behavioral audiometry, indicating differential recovery patterns by age.
- Baseline distortion product otoacoustic emission (DPOAE) signal-to-noise ratios correlated with improved ABR and ASSR thresholds at 26 weeks, suggesting baseline cochlear function may predict hearing recovery outcomes.
