The development of inner ear gene therapy for DFNB9: From bench to bedside

Hearing loss is a significant health problem facing the world’s population today. Approximately 3 in 1000 children born in the US are found to have hereditary hearing loss. Currently, hearing aids and cochlear implants represent the main treatment options for these patients. The field of gene replacement therapy as a treatment for hereditary hearing loss has advanced rapidly. There are now five ongoing clinical trials using gene replacement therapy to target non-syndromic autosomal recessive hearing loss DFNB9. The preliminary results from these studies are truly exciting. In this review, we summarize the preclinical and clinical studies that led to the development of these therapies, and discuss the potential challenges of applying gene replacement therapy for other types of hereditary hearing loss.