AAV2.7m8 is a powerful viral vector for inner ear gene therapy

Nat Commun. 2019 Jan 25;10(1):427. doi: 10.1038/s41467-018-08243-1.


Adeno-associated virus (AAV) has been successfully used to deliver gene therapy to improve auditory function in mouse models of hereditary hearing loss. Many forms of hereditary hearing loss have mutations which affect the cochlear hair cells, the mechanosensory cells which allow for sound detection and processing. While most conventional AAVs infect inner hair cells (IHCs) with various efficiencies, they infect outer hair cells (OHCs) and supporting cells at lower levels in the cochlea. Here we examine the infection patterns of two synthetic AAVs (AAV2.7m8 and AAV8BP2) in the mouse inner ear. AAV2.7m8 infects both IHCs and OHCs with high efficiency. In addition, AAV2.7m8 infects inner pillar cells and inner phalangeal cells with high efficiency. Our results suggest that AAV2.7m8 is an excellent viral vector for inner ear gene therapy targeting cochlear hair cells and supporting cells, and it will likely greatly expand the potential applications for inner ear gene therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Animals, Newborn
  • Dependovirus / genetics*
  • Dependovirus / metabolism
  • Disease Models, Animal
  • Gene Expression
  • Genes, Reporter
  • Genetic Therapy / methods*
  • Genetic Vectors / chemistry
  • Genetic Vectors / metabolism*
  • Green Fluorescent Proteins / genetics
  • Green Fluorescent Proteins / metabolism
  • Hair Cells, Auditory, Inner / metabolism*
  • Hair Cells, Auditory, Inner / pathology
  • Hair Cells, Auditory, Outer / metabolism
  • Hair Cells, Auditory, Outer / pathology
  • Hearing / genetics
  • Hearing Loss, Sensorineural / genetics
  • Hearing Loss, Sensorineural / metabolism
  • Hearing Loss, Sensorineural / pathology
  • Hearing Loss, Sensorineural / therapy*
  • Mice
  • Myosin VIIa
  • Myosins / genetics*
  • Myosins / metabolism


  • Myo7a protein, mouse
  • Myosin VIIa
  • Green Fluorescent Proteins
  • Myosins