title: Gene therapy for the inner ear.
abstract: Animal studies on inner ear development, repair and regeneration provide understanding of molecular pathways that can be harnessed for treating inner ear disease. Use of transgenic mouse technology, in particular, has contributed knowledge of genes that regulate development of hair cells and innervation, and of molecular players that can induce regeneration, but this technology is not applicable for human treatment, for practical and ethical reasons. Therefore other means for influencing gene expression in the inner ear are needed. We describe several gene vectors useful for inner ear gene therapy and the practical aspects of introducing these vectors into the ear. We then review the progress toward using gene transfer for therapies in both auditory and balance systems, and discuss the technological milestones needed to advance to clinical application of these methods.
authors.affiliation: Kresge Hearing Research Institute, Department of Otolaryngology, University of Michigan, 1150 West Medical Center Dr., Ann Arbor, MI 48109-5648, USA.
authors.forename: Hideto
authors.initials: H
authors.lastname: Fukui
authors.forename: Yehoash
authors.initials: Y
authors.lastname: Raphael
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D007758:Ear, Inner / Q000502:physiology*; D015870:Gene Expression; D020869:Gene Expression Profiling; D005786:Gene Expression Regulation; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006319:Hearing Loss, Sensorineural / Q000628:therapy*; D006801:Humans; D007759:Labyrinth Diseases / Q000628:therapy*; D051379:Mice; D008822:Mice, Transgenic; D009416:Nerve Regeneration; D009474:Neurons / Q000502:physiology; D015398:Signal Transduction