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遗传性听力损失的基因治疗:进展与尚存挑战

Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges.

作者信息

Hahn Roni, Avraham Karen B

机构信息

Department of Human Molecular Genetics and Biochemistry, Faculty of Medicine and Sagol School of Neuroscience, Tel Aviv University, Tel Aviv 6997801, Israel.

出版信息

Audiol Res. 2023 Dec 4;13(6):952-966. doi: 10.3390/audiolres13060083.

Abstract

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.

摘要

听力损失是人类中最普遍的感觉缺陷,构成了重大的全球健康挑战。预测表明,到2050年,世界上约10%的人口将面临致残性听力障碍。虽然约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。生物治疗的缺乏促使近年来人们付出了巨大努力,重点大力开展基因治疗以解决遗传性听力损失问题。尽管多项研究已在小鼠模型中显示出从常见形式的遗传性耳聋中有望恢复的效果,但要使基因治疗在不久的将来得以应用,仍必须克服现有挑战。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关键障碍。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/891f/10740825/930c921dc762/audiolres-13-00083-g001.jpg

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