期刊
CELL DEATH AND DIFFERENTIATION
卷 23, 期 8, 页码 1347-1357出版社
SPRINGERNATURE
DOI: 10.1038/cdd.2016.16
关键词
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资金
- National Basic Research Program of China [2012CB967902, 2014CB541705]
- National Development Program of Important Scientific Instruments [2013YQ030595]
- Chinese Academy of Sciences [XDA04020202-23]
- Opening Foundation of the State Key Laboratory of Space Medicine Fundamentals and Applications [SMFA12K02]
- TZ-1 Application Program [KYTZ01-0901-FB-003]
- Chinese National Science Foundation [81570932, 81330024]
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.4642G4 > A and c.8374G4 > 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.
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