title: Atoh1 gene therapy in the cochlea for hair cell regeneration.
abstract: BACKGROUND
  The sensory epithelium of the cochlea is a complex structure containing hair cells, supporting cells and auditory nerve endings, all of which degenerate after hearing loss in mammals. Biological approaches are being considered to preserve and restore the sensory epithelium after hearing loss. Of particular note is the ectopic expression of the Atoh1 gene, which has been shown to convert residual supporting cells into hair cells with restoration of function in some cases.
  
  
  METHODS
  In this review, hair cell development, spontaneous regeneration and hair cell regeneration mediated by Atoh1 gene therapy in the cochlea are discussed.
  
  
  CONCLUSIONS
  Gene therapy can be safely delivered locally to the inner ear and can be targeted to the sensory epithelium of the cochlea. Expression of the Atoh1 gene in supporting cells results in their transformation into cells with the appearance and function of immature hair cells but with the resulting loss of the original supporting cell. While the feasibility of Atoh1 gene therapy in the cochlea is largely dependent on the severity of the hearing loss, hearing restoration can be achieved in some situations. With further advances in Atoh1 gene therapy, hearing loss may not be as permanent as once thought.
authors: Bionics Institute , 384 Albert Street, East Melbourne, Victoria 3002 , Australia +613 9288 7594 ; +613 9667 7518 ; rrichardson@bionicsinstitute.org.
authors: Rachael T
authors: RT
authors: Richardson
authors: Patrick J
authors: PJ
authors: Atkinson
mesh_terms: D000818:Animals; D051792:Basic Helix-Loop-Helix Proteins / Q000008:administration & dosage / Q000235:genetics*; D003051:Cochlea / Q000502:physiology; D015316:Genetic Therapy / Q000379:methods*; D006198:Hair Cells, Auditory / Q000502:physiology*; D006801:Humans; D012038:Regeneration / Q000235:genetics*
chemical_list: D051792:Basic Helix-Loop-Helix Proteins; C500942:ATOH1 protein, human
keywords: adenovirus; atoh1; cochlea; gene therapy; hair cell; hearing loss; regeneration; supporting cell