Gene Therapy for the Inner Ear: Progress and Prospects

Abstract

Inner ear diseases increasingly affected the quality of life of patients. The current diagnostic protocol used in auditory area has been in place for over half a century and is insensitive to detect alterations in hearing status at early stage. Furthermore, no effective treatment is available for inner ear diseases although some patients may benefit from hearing aid with a hearing amplifier or cochlear implantation. Understanding the molecular mechanisms and investigating the fundamental process of hearing disorder could help us to avoid hearing defects or even to cure the disease by gene-based or molecular therapy. Currently, gene therapy for inner ear disease has become an emerging field of study. The medicine for treatment of inner ear diseases is undergoing a revolutionary change since the completion of the human genome sequencing. Various new discoveries and advanced technologies have been made in inner ear gene therapy such as viral-based or nonviral-based gene vectors, delivery strategies, and therapeutic genes and targets as well as the animal models for study of gene therapy for inner ear. Gene therapy may become an effective treatment in clinic for inner ear diseases in the future. This chapter is to summarize current significant advances and technological challenges for inner ear gene therapy and to discuss the previous works in detail by examining and analyzing the publications of inner ear gene therapy from the literature and patent documents and identify the promising methods, novel techniques, and vital research projects. Additionally, we also discuss the progress and prospects of inner ear gene therapy, the advances and shortages with possible solutions within this field of research.