title: [Gene therapy for human hearing loss: challenges and promises].
abstract: Thanks to the advances accomplished in human genomics during the last twenty years, major progress has been made towards understanding the pathogenesis of various forms of congenital or acquired deafness. The identification of deafness genes, which are potential therapeutic targets, and generation and functional characterization of murine models for human deafness forms have advanced the knowledge of the molecular physiology of auditory sensory cells. These milestones have opened the way for the development of new therapeutic strategies, alternatives to conventional prostheses, hearing amplification for mild-to-severe hearing loss, or cochlear implantation for severe-to-profound deafness. In this review, we first summarize the progress made over the last decade in using gene therapy and antisense RNA delivery, including the development of new methods for cochlear gene transfer. We then discuss the potential of gene therapy for curing acquired or inherited deafness and the major obstacles that must be overcome before clinical application can be considered.
authors.affiliation: Institut Pasteur, unité de génétique et physiologie de l'audition, 25, rue du Docteur Roux, 75724 Paris Cedex 15, France - Inserm UMRS 1120, 75015 Paris, France.
authors.forename: Anaïs
authors.initials: A
authors.lastname: Meyer
authors.forename: Christine
authors.initials: C
authors.lastname: Petit
authors.forename: Saaid
authors.initials: S
authors.lastname: Safieddine
mesh_terms: D000818:Animals; D007758:Ear, Inner / Q000378:metabolism; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D034381:Hearing Loss / Q000453:epidemiology / Q000235:genetics / Q000628:therapy*; D006801:Humans; D051379:Mice