CRISPR-Cas系统治疗遗传性听力损失的最新进展。

IF 1.5 Q4 CELL BIOLOGY American journal of stem cells Pub Date : 2023-08-15 eCollection Date: 2023-01-01
Ge Yin, Xiao-Hui Wang, Yu Sun
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引用次数: 0

摘要

遗传性听力损失已成为一个需要关注的重大公共卫生问题。在各种治疗策略中,基于基因编辑技术的基因治疗被认为是通过修复或消除突变基因来解决遗传性听力损失的最有前途的方法。集群规则间隔短回文重复序列(CRISPR)-Cas系统的出现通过其卓越的基因编辑能力彻底改变了基因治疗。该系统已广泛应用于哺乳动物基因编辑,目前正在通过临床试验进行评估。在此背景下,本综述旨在概述利用CRISPR-Cas系统治疗遗传性听力损失的最新进展。此外,我们深入探讨了该系统目前在解决遗传性听力损失方面的应用面临的主要挑战和前景。
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Recent advances in CRISPR-Cas system for the treatment of genetic hearing loss.

Genetic hearing loss has emerged as a significant public health concern that demands attention. Among the various treatment strategies, gene therapy based on gene editing technology is considered the most promising approach for addressing genetic hearing loss by repairing or eliminating mutated genes. The advent of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system has revolutionized gene therapy through its remarkable gene editing capabilities. This system has been extensively employed in mammalian gene editing and is currently being evaluated through clinical trials. Against this backdrop, this review aims to provide an overview of recent advances in utilizing the CRISPR-Cas system to treat genetic hearing loss. Additionally, we delve into the primary challenges and prospects associated with the current application of this system in addressing genetic hearing loss.

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