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Progress in Modeling and Targeting Inner Ear Disorders with Pluripotent Stem Cells.
Stem Cell Reports ( IF 5.9 ) Pub Date : 2020-05-21 , DOI: 10.1016/j.stemcr.2020.04.008
Pei-Ciao Tang 1 , Eri Hashino 2 , Rick F Nelson 1
Affiliation  

Sensorineural hearing loss and vestibular dysfunction are caused by damage to neurons and mechanosensitive hair cells, which do not regenerate to any clinically relevant extent in humans. Several protocols have been devised to direct pluripotent stem cells (PSCs) into inner ear hair cells and neurons, which display many properties of their native counterparts. The efficiency, reproducibility, and scalability of these protocols are enhanced by incorporating knowledge of inner ear development. Modeling human diseases in vitro through genetic manipulation of PSCs is already feasible, thereby permitting the elucidation of mechanistic understandings of a wide array of disease etiologies. Early studies on transplantation of PSC-derived otic progenitors have been successful in certain animal models, yet restoration of function and long-term cell survival remain unrealized. Through further research, PSC-based approaches will continue to revolutionize our understanding of inner ear biology and contribute to the development of therapeutic treatments for inner ear disorders.



中文翻译:

多能干细胞在建模和靶向内耳疾病方面的进展。

感觉神经性听力减退和前庭功能障碍是由神经元和机械敏感性毛细胞的损害引起的,这些损害在人类中不会再生到任何临床相关程度。已经设计了几种方案来将多能干细胞(PSC)引导到内耳毛细胞和神经元中,这些细胞显示出其天然对应物的许多特性。通过整合内耳发育知识,可以提高这些协议的效率,可重复性和可扩展性。体外模拟人类疾病通过对PSC进行基因操作已经是可行的,从而可以阐明对各种疾病病因的机理理解。在某些动物模型中,PSC来源的耳祖细胞移植的早期研究已经成功,但是功能恢复和长期细胞存活仍未实现。通过进一步的研究,基于PSC的方法将继续革新我们对内耳生物学的理解,并为内耳疾病的治疗方法的发展做出贡献。

更新日期:2020-05-21
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