AAV-mediated rescue of <i>Eps8</i> expression <i>in vivo</i> restores hair-cell function in a mouse model of recessive deafness.

ABSTRACT: The transduction of acoustic information by hair cells depends upon mechanosensitive stereociliary bundles that project from their apical surface. Mutations or absence of the stereociliary protein EPS8 cause deafness in humans and mice, respectively. Eps8 knockout mice (Eps8 ^-/- ) have hair cells with immature stereocilia and fail to become sensory receptors. Here, we show that exogenous delivery of Eps8 using Anc80L65 in P1-P2 Eps8 ^-/- mice in vivo rescued the hair bundle structure of apical-coil hair cells. Rescued hair bundles correctly localize EPS8, WHIRLIN, MYO15, and BAIAP2L2, and generate normal mechanoelectrical transducer currents. Inner hair cells with normal-looking stereocilia re-expressed adult-like basola

SUBMITTER: Jeng JY 

PROVIDER: S-EPMC9382420 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature