Inner ear target cells (AAV-mediated delivery)

The term "inner ear target cells via AAV capsid and cell-specific promoter" refers to a precision gene therapy delivery strategy rather than a single molecular target. This approach utilizes engineered Adeno-Associated Virus (AAV) vectors, such as Anc80L65 or AAV-S, designed to bypass physiological barriers and efficiently transduce specific cell types within the cochlea, such as inner hair cells (IHCs), outer hair cells (OHCs), or spiral ganglion neurons (SGNs) (Landegger et al., Nat Commun, 2017). To ensure cell-type specificity and reduce off-target effects, these vectors incorporate specialized promoters like Myo7a for hair cells or Gfap for supporting cells (Valero et al., Gene Ther, 2017). This methodology is primarily employed to treat genetic forms of sensorineural hearing loss, such as those caused by mutations in the OTOF, TMC1, or GJB2 genes (Omichi et al., Hum Gene Ther, 2019). By delivering a functional transgene directly to the affected cells, the therapy aims to restore sensory transduction and auditory function. Current clinical candidates using this delivery paradigm include AK-OTOF and DB-OTO for otoferlin-related deafness. Safety considerations include the potential for immune responses to the viral capsid and the invasive nature of sub-millimeter surgical delivery to the inner ear.

Other names

AAV-mediated inner ear gene therapyCochlear cell targetingInner ear cell-specific AAV deliveryAAV-mediated transduction of inner ear cells

Targeted delivery of genetic material to specific inner ear cell populations using viral capsids and promoters to restore protein expression or silence pathogenic genes.