Effects of genetic correction on the differentiation of hair cell-like cells from iPSCs with MYO15A mutation.

Effects of genetic correction on the differentiation of hair cell-like cells from iPSCs with MYO15A mutation.

Cell Death Differ. 2016 Feb 26;

Authors: Chen JR, Tang ZH, Zheng J, Shi HS, Ding J, Qian XD, Zhang C, Chen JL, Wang CC, Li L, Chen JZ, Yin SK, Shao JZ, Huang TS, Chen P, Guan MX, Wang JF

Abstract
Deafness or hearing loss is a major issue in human health. Inner ear hair cells are the main sensory receptors responsible for hearing. Defects in hair cells are one of the major causes of deafness. A combination of induced pluripotent stem cell (iPSC) technology with genome-editing technology may provide an attractive cell-based strategy to regenerate hair cells and treat hereditary deafness in humans. Here, we report the generation of iPSCs from members of a Chinese family carrying MYO15A c.4642G>A and c.8374G>A mutations and the induction of hair cell-like cells from those iPSCs. The compound heterozygous MYO15A mutations resulted in abnormal morphology and dysfunction of the derived hair cell-like cells. We used a CRISPR/Cas9 approach to genetically correct the MYO15A mutation in the iPSCs and rescued the morphology and function of the derived hair cell-like cells. Our data demonstrate the feasibility of generating inner ear hair cells from human iPSCs and the functional rescue of gene mutation-based deafness by using genetic correction.Cell Death and Differentiation advance online publication, 26 February 2016; doi:10.1038/cdd.2016.16.

PMID: 26915297 [PubMed - as supplied by publisher]

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