遗传性听力损失的基因疗法:最新进展和依然存在的挑战。

IF 2.1 Q1 AUDIOLOGY & SPEECH-LANGUAGE PATHOLOGY
Roni Hahn, Karen B Avraham
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引用次数: 0

摘要

听力损失是人类最普遍的感官缺陷,对全球健康构成重大挑战。据预测,到 2050 年,全球约有 10% 的人口将面临听力障碍的困扰。虽然约有一半的先天性听力损失病例有遗传病因,但助听器和人工耳蜗等传统干预措施并不能完全恢复正常听力。近年来,由于缺乏生物治疗方法,人们开始大力发展基因疗法,以解决遗传性听力损失问题。虽然一些研究显示,小鼠模型中常见形式的遗传性耳聋有望得到康复,但要在不久的将来应用基因疗法,还必须克服现有的挑战。在此,我们总结了过去几年采用的主要基因治疗策略,概述了遗传性听力损失临床前研究的最新成果,并概述了当前耳蜗基因治疗的主要障碍。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges.

Hearing loss stands as the most prevalent sensory deficit among humans, posing a significant global health challenge. Projections indicate that by 2050, approximately 10% of the world's population will grapple with disabling hearing impairment. While approximately half of congenital hearing loss cases have a genetic etiology, traditional interventions such as hearing aids and cochlear implants do not completely restore normal hearing. The absence of biological treatment has prompted significant efforts in recent years, with a strong focus on gene therapy to address hereditary hearing loss. Although several studies have exhibited promising recovery from common forms of genetic deafness in mouse models, existing challenges must be overcome to make gene therapy applicable in the near future. Herein, we summarize the primary gene therapy strategies employed over past years, provide an overview of the recent achievements in preclinical studies for genetic hearing loss, and outline the current key obstacles to cochlear gene therapy.

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来源期刊
Audiology Research
Audiology Research AUDIOLOGY & SPEECH-LANGUAGE PATHOLOGY-
CiteScore
2.30
自引率
23.50%
发文量
56
审稿时长
11 weeks
期刊介绍: The mission of Audiology Research is to publish contemporary, ethical, clinically relevant scientific researches related to the basic science and clinical aspects of the auditory and vestibular system and diseases of the ear that can be used by clinicians, scientists and specialists to improve understanding and treatment of patients with audiological and neurotological disorders.
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