Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Gene Therapy for Inherited Hearing Loss: Updates and Chal-lenges

Version 1 : Received: 21 October 2023 / Approved: 23 October 2023 / Online: 23 October 2023 (10:52:29 CEST)

A peer-reviewed article of this Preprint also exists.

Hahn, R.; Avraham, K.B. Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges. Audiol. Res. 2023, 13, 952-966. Hahn, R.; Avraham, K.B. Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges. Audiol. Res. 2023, 13, 952-966.

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 these 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 of 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 the preclinical studies for genetic hearing loss, and outline the current key obstacles to cochlear gene therapy.

Keywords

genetics; genomics; deafness; inner ear; cochlea; delivery; editing

Subject

Medicine and Pharmacology, Otolaryngology

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