Cabp2-Gene Therapy Restores Inner Hair Cell Calcium Currents and Improves Hearing in a DFNB93 Mouse Model.

ABSTRACT: Clinical management of auditory synaptopathies like other genetic hearing disorders is currently limited to the use of hearing aids or cochlear implants. However, future gene therapy promises restoration of hearing in selected forms of monogenic hearing impairment, in which cochlear morphology is preserved over a time window that enables intervention. This includes non-syndromic autosomal recessive hearing impairment DFNB93, caused by defects in the CABP2 gene. Calcium-binding protein 2 (CaBP2) is a potent modulator of inner hair cell (IHC) voltage-gated calcium channels Ca_V1.3. Based on disease modeling in Cabp2^-/- mice, DFNB93 hearing impairment has been ascribed to enhanced steady-state inactivation of IHC Ca_V1.3 channels, effectively limiting the

SUBMITTER: Oestreicher D 

PROVIDER: S-EPMC8417311 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature