Gene Editing Technologies for Hereditary Hearing Loss: Prospects and Challenges

Area of Science:

• Genetics
• Molecular Biology
• Otolaryngology

Background:

• Hereditary deafness is a major global health issue with few treatments.
• Monogenic mutations cause most hereditary deafness, making them targets for gene editing.

Purpose of the Study:

• To review advancements in gene editing platforms for hereditary deafness.
• To assess the potential benefits and challenges of clinical applications for congenital hearing impairment.

Main Methods:

• Review of recent gene editing technologies: engineered nucleases, base editors (BEs), prime editors (PEs), and mitochondrial editors.
• Evaluation of efficacy in preclinical mouse models of hereditary deafness.
• Analysis of genetic heterogeneity and clinical translation hurdles.

Main Results:

• Gene editing tools like BEs and PEs show efficacy in mouse models for correcting deafness-causing mutations.
• Significant progress has been made in precise genomic modification for hearing restoration.
• Early-stage research indicates potential but highlights the need for further development.

Conclusions:

• Gene editing offers a promising therapeutic avenue for hereditary deafness.
• Overcoming genetic heterogeneity and technical challenges is crucial for clinical translation.
• Further research is needed to realize the full potential of gene editing for congenital hearing impairment.