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A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome.
Frontiers in Cellular Neuroscience ( IF 5.3 ) Pub Date : 2020-05-28 , DOI: 10.3389/fncel.2020.00183
Lucy S French 1 , Carla B Mellough 1 , Fred K Chen 1, 2, 3 , Livia S Carvalho 1
Affiliation  

Usher syndrome is a genetic disorder causing neurosensory hearing loss and blindness from retinitis pigmentosa (RP). Adaptive techniques such as braille, digital and optical magnifiers, mobility training, cochlear implants, or other assistive listening devices are indispensable for reducing disability. However, there is currently no treatment to reduce or arrest sensory cell degeneration. There are several classes of treatments for Usher syndrome being investigated. The present article reviews the progress this research has made towards delivering commercial options for patients with Usher syndrome.



中文翻译:

亚瑟氏综合症基因,药物和细胞疗法的综述。

Usher综合征是一种遗传性疾病,可导致色素性视网膜炎(RP)导致神经感觉性听力减退和失明。盲文,数字和光学放大镜,行动能力训练,人工耳蜗或其他辅助聆听设备等自适应技术对于减少残疾是必不可少的。但是,目前尚无减少或阻止感觉细胞变性的治疗方法。正在研究针对Usher综合征的几种治疗方法。本文回顾了这项研究在为Usher综合征患者提供商业选择方面取得的进展。

更新日期:2020-07-09
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