Gadenstaetter 2024 — Inner Ear Gene Therapy: Bench to Bedside Overview
Citation: Gadenstaetter AJ, et al. (2024). “Inner Ear Gene Therapy: An Overview from Bench to Bedside.” Molecular Diagnosis & Therapy, doi:10.1007/s40291-024-00759-1.
File: ~/BookLibrary/incoming/Gadenstaetter_2024_Inner-ear-gene-therapy-bench-bedside_MolDiagTher.pdf
Coverage
Comprehensive review of state-of-the-art inner ear gene therapy as of late 2024:
- Delivery methods: round window, posterior semicircular canal, endolymphatic sac
- Vector comparison: AAV serotypes, LNP, viral vs non-viral
- Target genes: OTOF (otoferlin, DFNB9), GJB2 (connexin 26), STRC (DFNB16), CDH23, MYO7A
- Clinical trials: OTOF trials (Regeneron, Akouos, Shanghai) — first human data
- Safety: immune response, cochleotoxicity, off-target expression
Key Data Points
- OTOF gene therapy (DFNB9): first clinical results 2023-2024 showing partial hearing recovery in children → proof that inner ear gene therapy works in humans
- STRC section: described as high-priority target, dual-vector approach validated in mice
- First clinical trials expected for STRC: 2026-2028 window
- Anc80L65 and AAV2/1 remain gold standard for cochlear OHC transduction
Relevance
Good overview to understand where STRC fits in the broader gene therapy landscape. OTOF success paves the regulatory and clinical path for STRC.
Connections
- STRC Gene Therapy Landscape 2026 [see-also]
- Iranfar 2026 — Dual AAV STRC [see-also]
- Misha [applies]
- Jeffrey Holt [see-also]