Challenges and Strategies for Accelerating the Clinical Translation of Gene Therapy for Hearing and Balance Disorders

Hearing loss and balance disorders affect a large number of individuals worldwide and can have substantial impacts on communication, development, mobility, and quality of life. Recent advances in molecular genetics, vector engineering, inner ear delivery techniques, and disease modeling have accelerated the development of gene therapy approaches for these conditions. At the same time, the field has entered a stage in which clinical translation is becoming increasingly realistic, with several programs moving closer to or into human studies. Despite this progress, key challenges remain, including target selection, treatment timing, delivery efficiency, durability, safety, outcome assessment, and the translation of preclinical findings into meaningful clinical benefit.Gene therapy for hearing and balance disorders is moving from experimental research toward clinical application. Early translational and clinical efforts have demonstrated the promise of gene-based approaches for treating inner ear diseases, while also revealing important scientific, technical, and clinical barriers that must be addressed before these therapies can be more broadly and efficiently implemented. This Research Topic aims to identify the major challenges currently limiting clinical translation and to highlight strategies that may help overcome them. We welcome contributions spanning basic, preclinical, translational, and clinical research that can advance the safe, effective, and timely development of gene therapy for auditory and vestibular disorders.This Research Topic will focus on the challenges and emerging solutions involved in accelerating the clinical translation of gene therapy for hearing and balance disorders. We invite submissions addressing, but not limited to, vector design, cargo optimization, inner ear delivery approaches, treatment windows, animal and disease models, biomarkers, outcome measures, safety evaluation, immunological considerations, manufacturing issues, regulatory perspectives, and early clinical or translational studies. Manuscripts that bridge basic science and clinical application are especially encouraged. We welcome Original Research, Review, Mini Review, Methods, Perspective, and Clinical Study articles that provide insight into how gene therapy can be more effectively advanced toward clinical use in audiology and otology. Topic editors: Nesrine Benkafadar, Masao Noda Source: https://www.frontiersin.org/research-topics/81382/challenges-and-strategies-for-accelerating-the-clinical-translation-of-gene-therapy-for-hearing-and-balance-disorders