Cited by 333Virus-based gene therapy researchHearing, Cochlea, Tinnitus, GeneticsRNA Interference and Gene Delivery

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A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

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Landegger, Lukas D, Pan, Bifeng, Askew, Charles, Wassmer, Sarah J, Gluck, Sarah D, Galvin, Alice, Taylor, Ruth, Forge, Andrew, Stankovic, Konstantina M, Holt, Jeffrey R, Vandenberghe, Luk H·2017·Nature Biotechnology

DOI 10.1038/nbt.3781PMC PMC5340646

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Abstract

Efforts to develop gene therapies for hearing loss have been hampered by the lack of safe, efficient, and clinically relevant delivery modalities. Here we demonstrate the safety and efficiency of Anc80L65, a rationally designed synthetic vector, for transgene delivery to the mouse cochlea. Ex vivo transduction of mouse organotypic explants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell targets. Round window membrane injection resulted in highly efficient transduction of inner and outer hair cells in mice, a substantial improvement over conventional AAV vectors. Anc80L65 round window injection was well tolerated, as indicated by sensory cell function, hearing and vestibular function, and immunologic parameters. The ability of Anc80L65 to target outer hair cells at high rates, a requirement for restoration of complex auditory function, may enable future gene therapies for hearing and balance disorders.

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Authors: Landegger, L. D., Pan, B., Askew, C., Wassmer, S. J., Gluck, S. D., Galvin, A., Taylor, R., Forge, A., Stankovic, K. M., Holt, J. R., & Vandenberghe, L. H.
Affiliations: Massachusetts Eye and Ear Infirmary (US)
Boston Children's Hospital (US)
University College London (GB)
Corresponding author: Holt, Jeffrey R
Vandenberghe, Luk H
ORCID: Landegger, Lukas D
Pan, Bifeng
Askew, Charles
Galvin, Alice
Taylor, Ruth
Forge, Andrew
Holt, Jeffrey R
Vandenberghe, Luk H
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2017-02-06
Volume: 35
Issue: 3
Pages: 280-284
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt.3781
PMC: PMC5340646
OpenAlex: W2586762515
Cited by: 325
References: 39
Citations by year: 2026: 27 · 2025: 28 · 2024: 30 · 2023: 33 · 2022: 39 · 2021: 34 · 2020: 53 · 2019: 30 · 2018: 31 · 2017: 20
FWCI: 23.76
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springernature.com/gp/researchers/text-and-data-mining (tdm)
www.springernature.com/gp/researchers/text-and-data-mining (vor)
Locations: Nature Biotechnology (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Carolina Digital Repository (University of North Carolina at Chapel Hill) (submittedVersion)
Digital Access to Scholarship at Harvard (DASH) (Harvard University) (submittedVersion, cc-by)
UCL Discovery (University College London) (submittedVersion)
UNC Libraries
Funders: U.S. Department of Defense (W81XWH-15–1-, W81XWH, W81XWH1510472)
Fondation Bertarelli
Grousbeck Family Foundation
Rosetrees Trust (M58-F1)
National Institutes of Health (1dp1od008267-01, W81XWH)
National Eye Institute (DP1EY023177)
National Institute on Deafness and Other Communication Disorders (R01DC015824)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (U54HD090255)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Neuroscience › Sensory Systems › Hearing, Cochlea, Tinnitus, Genetics
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords: adeno-associated virus vectors, inner hair cells, outer hair cells, vestibular function, hearing restoration, balance disorders
MeSH terms: Dependovirus, Animals, Cochlea, Genetic Vectors, Mice, Inbred C57BL, Plasmids, Transduction, Genetic, Genetic Therapy, Mice

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A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

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