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The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness
Jiao Zhou1, Di Deng2, Chunmei Gan3
1Medicine and Engineering Interdisciplinary Research Laboratory of Nursing and Materials, West China Hospital, Sichuan University/West China School of Nursing, Sichuan University Chengdu 610041, Sichuan, P. R. China.
Insights
Gene therapy shows promise for hereditary hearing loss (HHL), a widespread condition. Overcoming challenges in gene delivery and safety is crucial for developing effective treatments for genetic deafness.
Area of Science:
- Otolaryngology
- Genetics
- Molecular Biology
Background:
- Hereditary hearing loss (HHL) affects over 466 million people globally.
- HHL is categorized into syndromic (SHL) and nonsyndromic (NSHL) forms, with no current cure for genetic causes.
- Existing treatments like hearing aids and cochlear implants manage symptoms, not the underlying genetic defects.
Purpose of the Study:
- To systematically review the progress of gene therapy for hereditary hearing loss.
- To evaluate the barriers hindering the clinical implementation of gene therapy for HHL.
- To provide insights for future translational research in genetic deafness.
Main Methods:
- Systematic literature review of gene therapy studies for hereditary hearing loss.
- Analysis of current research on gene therapy approaches and their efficacy in animal models.
- Evaluation of challenges related to viral vector safety, transfection efficiency, and target specificity.
Main Results:
- Gene therapy has demonstrated therapeutic potential in preclinical models of genetic deafness.
- Significant challenges remain for clinical translation, including optimizing vector delivery and ensuring safety.
- Current research highlights the need for improved specificity and efficiency in gene targeting.
Conclusions:
- Gene therapy represents a promising avenue for treating hereditary hearing loss.
- Addressing safety, efficiency, and specificity barriers is essential for successful clinical application.
- Further translational research is required to bring gene therapy from animal models to human patients.
Abstract:
Hearing loss is a prevalent organ-specific disorder affecting individuals throughout their lifespan, with over 466 million cases reported globally. The conditions can be classified into two broad categories: hereditary and nonhereditary. HHL, caused by genetic mutations or chromosomal abnormalities, can be divided into nonsyndromic (NSHL) and syndromic (SHL) subtypes. NSHL presents as isolated auditory impairment without systemic manifestations, whereas SHL involves concurrent dysfunction in other organ systems. Nonhereditary hearing loss typically results from infections, ototoxic drugs, noise exposure, trauma, or age-related degeneration. Current clinical interventions focus on symptom management through hearing aids and cochlear implants, as no curative treatment exists for genetic forms. Recent studies have shown the therapeutic potential of gene therapy in animal models of genetic deafness, although clinical translation faces challenges, including viral vector safety, transfection efficiency, and target specificity. This systematic review synthesizes current progress in gene therapy for HHL and evaluates barriers to clinical implementation, offering insights for future translational studies.
