Recent development of AAV-based gene therapies for inner ear disorders

Gene Ther. 2020 Aug;27(7-8):329-337. doi: 10.1038/s41434-020-0155-7. Epub 2020 May 18.

Abstract

Gene therapy for auditory diseases is gradually maturing. Recent progress in gene therapy treatments for genetic and acquired hearing loss has demonstrated the feasibility in animal models. However, a number of hurdles, such as lack of safe viral vector with high efficiency and specificity, robust deafness large animal models, translating animal studies to clinic etc., still remain to be solved. It is necessary to overcome these challenges in order to effectively recover auditory function in human patients. Here, we review the progress made in our group, especially our efforts to make more effective and cell type-specific viral vectors for targeting cochlea cells.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cochlea
  • Dependovirus* / genetics
  • Genetic Therapy*
  • Genetic Vectors / genetics
  • Hearing Loss* / genetics
  • Hearing Loss* / therapy
  • Humans