Omichi 2020 — Hair Cell Transduction Efficiency of Single- and Dual-AAV
Citation: Omichi R, Yoshimura H, Shibata SB, Vandenberghe LH, Smith RJH. (2020). “Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae.” Molecular Therapy: Methods & Clinical Development, 17, 1167–1177.
DOI: 10.1016/j.omtm.2020.05.007
PMC: PMC7270144
File: ~/BookLibrary/incoming/Omichi_2020_Hair-cell-transduction-dual-single-AAV-cochlea_MolTherMethods.pdf
Key Findings
- Comprehensive comparison of AAV serotypes for cochlear hair cell transduction in adult mice
- Tested: AAV1, AAV2, AAV5, AAV6, AAV8, AAV9, Anc80L65, AAV2/Anc80L65
- Anc80L65 = highest transduction efficiency in both IHC and OHC
- Dual-AAV systems (for large genes like STRC): AAV2/Anc80L65 + AAV2/Anc80L65 works well
- Adult cochlea is harder to transduce than neonatal — but Anc80L65 retains efficacy
Serotype Ranking (OHC transduction)
- Anc80L65 — ~85-90% OHC, ~95% IHC
- AAV2/PHP.eB — good but variable
- AAV9 — moderate
- AAV1, AAV8 — lower OHC efficiency
Delivery Routes Tested
- Round window membrane (RWM) injection — most practical, clinical route
- Posterior semicircular canal (PSC) — more consistent distribution
Relevance to STRC Gene Therapy
- STRC requires dual-AAV → Anc80L65 is the top choice for both vectors
- Adult transduction matters for human patients (not just neonatal mice)
- Efficiency data directly informs clinical trial design for DFNB16
Connections
- Iranfar 2026 — Dual AAV STRC [see-also]
- STRC Gene Therapy Landscape 2026 [source]
- Dual Vector AAV Strategy [source]
- Misha [applies]