Gene therapy in the inner ear: Mechanisms and clinical implications

Thomas R. Van De Water, Hinrich Staecker, Marc W. Halterman, Howard J. Federoff

Research output: Contribution to journalArticlepeer-review

32 Scopus citations


The application of gene therapy to the inner ear is an emerging field of study. Most studies report the expression of marker genes (e.g., galactosidase) within the tissues of the cochlea. The first biologic response of an inner ear tissue (i.e., auditory neurons) to transduction by a gene therapy vector expressing a therapeutic gene (a herpes amplicon vector containing a BDNF gene) was observed in spiral explants obtained from early postnatal rat cochleae. This study was important because it demonstrated the feasibility of a gene augmentation approach to treat traumatized cochleae. Long-term expression of transduced or transfected genes in cochlear tissues have been obtained with adenovirus, adeno-associated virus, and herpes amplicon vectors. The herpes amplicon vector (i.e., HSVbdnflac) that evoked a biologic response in vitro has also been successfully used to support the survival of auditory neurons in vivo following loss of the auditory hair cells (i.e., loss of trophic factor). Gene therapy has been successfully applied to the cochlea of a laboratory animal, and future studies will define the types of vectors and therapeutic genes that will work best for the treatment of inner ear diseases in the clinic.

Original languageEnglish (US)
Pages (from-to)345-360
Number of pages16
JournalAnnals of the New York Academy of Sciences
StatePublished - 1999
Externally publishedYes

ASJC Scopus subject areas

  • Neuroscience(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • History and Philosophy of Science


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