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

Kevin Isgrig, Devin S. McDougald, Jianliang Zhu, Hong Jun Wang, Jean Bennett, Wade W Chien

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish (US)
Article number427
JournalNature Communications
Volume10
Issue number1
DOIs
StatePublished - Dec 1 2019

Fingerprint

gene therapy
Gene therapy
hair
ear
Inner Ear
Genetic Therapy
Outer Auditory Hair Cells
Inner Auditory Hair Cells
Cells
Auditory Hair Cells
Audition
cells
auditory defects
Hearing Loss
mice
Dependovirus
Viruses
Gene Targeting
Cochlea
cochlea

ASJC Scopus subject areas

  • Chemistry(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Physics and Astronomy(all)

Cite this

AAV2.7m8 is a powerful viral vector for inner ear gene therapy. / Isgrig, Kevin; McDougald, Devin S.; Zhu, Jianliang; Wang, Hong Jun; Bennett, Jean; Chien, Wade W.

In: Nature Communications, Vol. 10, No. 1, 427, 01.12.2019.

Research output: Contribution to journalArticle

Isgrig, Kevin ; McDougald, Devin S. ; Zhu, Jianliang ; Wang, Hong Jun ; Bennett, Jean ; Chien, Wade W. / AAV2.7m8 is a powerful viral vector for inner ear gene therapy. In: Nature Communications. 2019 ; Vol. 10, No. 1.
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