The genetic correction of induced pluripotent stem cells (iPSCs) induced from 
somatic cells of patients with sensorineural hearing loss (caused by hereditary 
factors) is a promising method for its treatment. The correction of gene 
mutations in iPSCs could restore the normal function of cells and provide a rich 
source of cells for transplantation. In the present study, iPSCs were generated 
from a deaf patient with compound heterozygous MYO7A mutations (c.1184G>A and 
c.4118C>T; P-iPSCs), the asymptomatic father of the patient (MYO7A c.1184G>A 
mutation; CF-iPSCs), and a normal donor (MYO7A(WT/WT); C-iPSCs). One of MYO7A 
mutation sites (c.4118C>T) in the P-iPSCs was corrected using CRISPR/Cas9. The 
corrected iPSCs (CP-iPSCs) retained cell pluripotency and normal karyotypes. 
Hair cell-like cells induced from CP-iPSCs showed restored organization of 
stereocilia-like protrusions; moreover, the electrophysiological function of 
these cells was similar to that of cells induced from C-iPSCs and CF-iPSCs. 
These results might facilitate the development of iPSC-based gene therapy for 
genetic disorders.
SIGNIFICANCE: Induced pluripotent stem cells (iPSCs) were generated from a deaf 
patient with compound heterozygous MYO7A mutations (c.1184G>A and c.4118C>T). 
One of the MYO7A mutation sites (c.4118C>T) in the iPSCs was corrected using 
CRISPR/Cas9. The genetic correction of MYO7A mutation resulted in morphologic 
and functional recovery of hair cell-like cells derived from iPSCs. These 
findings confirm the hypothesis that MYO7A plays an important role in the 
assembly of stereocilia into stereociliary bundles. Thus, the present study 
might provide further insight into the pathogenesis of sensorineural hearing 
loss and facilitate the development of therapeutic strategies against monogenic 
disease through the genetic repair of patient-specific iPSCs.