What they found
TL;DR (mlx-community/Qwen3.5-35B-A3B-4bit): Researchers combined the AAV2.7m8 vector with the Myo15 promoter to achieve high-efficiency and safe gene delivery to outer hair cells in adult mice. This method successfully overcomes the previous bottleneck of low transduction rates in adult mammals using two different injection techniques.
Adeno-associated virus vector-mediated inner ear gene therapy has gradually achieved successful clinical translation in individual treatment regimens. However, the low transduction efficiency of AAV vectors in the outer hair cells of adult mammals has hindered the development and application of inner ear gene therapy strategies for hearing loss. In this study, we innovatively combined the AAV2.7m8 vector with the Myo15 promoter, achieving high-efficiency transduction of the outer hair cells in adult mice across different age groups. Subsequently, we compared the results of two gene delivery methods, posterior semicircular canal injection and round window membrane injection, in adult mice. Ultimately, we successfully established a highly efficient and targeted inner ear gene delivery system by combining AAV2.7m8 with the Myo15 promoter, which effectively resolves the bottleneck of low outer hair cell transduction efficiency in adult mice. This system not only achieves high-efficiency transduction of the outer hair cells across adult mice of different ages but also demonstrates the safety of local administration, as assessed through histopathological assessments of systemic organs and inner ear tissues. This optimized delivery strategy provides a reliable technical basis for the future clinical translation of inner ear gene therapy and the development of therapeutic regimens for sensorineural hearing loss.
How this applies to our program
This study provides a critical delivery mechanism for STRC/DFNB16 gene therapy by enabling efficient targeting of outer hair cells in adult mice, which is essential for treating hearing loss where these cells are damaged. The demonstrated safety and age-independent efficacy suggest this vector-promoter combination could be adapted for delivering STRC to adult patients.
Key numbers
- AAV2.7m8 vector combined with Myo15 promoter
- two gene delivery methods compared (posterior semicircular canal and round window membrane)
- successful transduction across different age groups of adult mice
Links
- pubmed_id: https://pubmed.ncbi.nlm.nih.gov/42478043/
- DOI: https://doi.org/10.1016/j.ymthe.2026.07.040
Connections
[source]auto-indexed 2026-08-05 by strc-lit-watch