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A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear
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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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“Later, more studies further characterized the vector Anc80L65, demonstrating great transduction efficiency for retinas [71], inner ear [72], and even notable expression in the CNS after systemic gene transfer [73].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [72]
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Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1108046 · Cited by 1,864
Zuris, J. A., Thompson, D. B., Shu, Y., Guilinger, J. P., Bessen, J. L., Hu, J. H., Maeder, M. L., Joung, J. K., Chen, Z.-Y., & Liu, D. R. (2015) · Nature Biotechnology · doi:10.1038/nbt.3081 · Cited by 1,460
MacLaren, R. E., Groppe, M., Barnard, A. R., Cottriall, C. L., Tolmachova, T., Seymour, L., Clark, K. R., During, M. J., Cremers, F. P. M., Black, G. C. M., et al. (2014) · The Lancet · doi:10.1016/s0140-6736(13)62117-0 · Cited by 796
Age-Related Cochlear Synaptopathy: An Early-Onset Contributor to Auditory Functional Decline
Sergeyenko, Y., Lall, K., Liberman, M. C., & Kujawa, S. G. (2013) · Journal of Neuroscience · doi:10.1523/jneurosci.1783-13.2013 · Cited by 794
Auditory hair cell replacement and hearing improvement by Atoh1 gene therapy in deaf mammals
Izumikawa, M., Minoda, R., Kawamoto, K., Abrashkin, K. A., Swiderski, D. L., Dolan, D. F., Brough, D. E., & Raphael, Y. (2005) · Nature Medicine · doi:10.1038/nm1193 · Cited by 742
Mammalian cochlear supporting cells can divide and trans-differentiate into hair cells
White, P. M., Doetzlhofer, A., Lee, Y. S., Groves, A. K., & Segil, N. (2006) · Nature · doi:10.1038/nature04849 · Cited by 419
Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally Mediated Gene Therapy
Akil, O., Seal, R. P., Burke, K., Wang, C., Alemi, A. S., During, M. J., Edwards, R. H., & Lustig, L. R. (2012) · Neuron · doi:10.1016/j.neuron.2012.05.019 · Cited by 402
Behavioral characterization of a unilateral 6-OHDA-lesion model of Parkinson's disease in mice
Ferfoglia, R. I., Mohapel, P., Brundin, P., & Paul, G. (2005) · Behavioural Brain Research · doi:10.1016/j.bbr.2005.02.023 · Cited by 387
In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector
Zinn, E., Pacouret, S., Khaychuk, V., Turunen, H. T., Carvalho, L. S., Andres-Mateos, E., Shah, S., Shelke, R., Maurer, A. C., Plovie, E., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.019 · Cited by 310
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Al‐Moyed, H., Cepeda, A. P., Jung, S., Moser, T., Kügler, S., & Reisinger, E. (2019) · EMBO Molecular Medicine · doi:10.15252/emmm.201809396 · Cited by 230
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Wang, J., & Puel, J. (2018) · Physiological Reviews · doi:10.1152/physrev.00053.2017 · Cited by 150
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Yoshimura, H., Shibata, S. B., Ranum, P. T., & Smith, R. J. (2018) · Scientific Reports · doi:10.1038/s41598-018-21233-z · Cited by 127
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Self-citationWesthaus, A., Cabanes-Creus, M., Rybicki, A., Baltazar, G., Navarro, R. G., Zhu, E., Drouyer, M., Knight, M., Albu, R. F., Ng, B. H., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.264 · Cited by 102
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Nikolić, S., Gazdic, M., Rosic, G. L., Kovačević, M. M., Jovicic, N. U., Nestorović, N. M., Stojković, P., Filipović, N., Milošević-Djordjević, O., Selaković, D., et al. (2022) · Environmental Pollution · doi:10.1016/j.envpol.2022.119206 · Cited by 87
Suzuki, J., Hashimoto, K., Xiao, R., Vandenberghe, L. H., & Liberman, M. C. (2017) · Scientific Reports · doi:10.1038/srep46827 · Cited by 85
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Self-citationTao, Y., Huang, M., Shu, Y., Ruprecht, A., Wang, H., Tang, Y., Vandenberghe, L. H., Wang, Q., Gao, G., Kong, W., et al. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.120 · Cited by 81
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Tian, Y., Zhou, M., Shi, H., Gao, S., Xie, G., Zhu, M., Wu, M., Chen, J., & Niu, Z. (2018) · Nano Letters · doi:10.1021/acs.nanolett.8b01805 · Cited by 70
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Noh, B., Rim, J. H., Gopalappa, R., Lin, H., Kim, K. M., Kang, M. J., Gee, H. Y., Choi, J. Y., Kim, S., & Jung, J. (2022) · Theranostics · doi:10.7150/thno.67781 · Cited by 60
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Carpena, N. T., & Lee, M. Y. (2018) · Genomics & Informatics · doi:10.5808/gi.2018.16.4.e20 · Cited by 51
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Gu, X. G., Wang, D., Xu, Z., Wang, J., Guo, L., Chai, R., Li, G., Shu, Y., & Li, H. (2021) · Genome biology · doi:10.1186/s13059-021-02311-4 · Cited by 51
Cui, C., Wang, D., Huang, B., Wang, F., Chen, Y., Lv, J., Zhang, L., Han, L., Liu, D., Chen, Z., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.07.016 · Cited by 50
Landegger, L. D., Vasilijić, S., Fujita, T., Soares, V. Y. R., Seist, R., Xu, L., & Stanković, K. M. (2019) · Frontiers in Neurology · doi:10.3389/fneur.2019.00977 · Cited by 50
Self-citationTalaei, S., Schnee, M. E., Aaron, K. A., & Ricci, A. J. (2019) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2019.00471 · Cited by 50
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Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48
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Du, T., Dewey, J. B., Wagner, E. L., Cui, R., Heo, J., Park, J.-J., Francis, S. P., Perez‐Reyes, E., Guillot, S. J., Sherman, N. E., et al. (2019) · Nature Communications · doi:10.1038/s41467-019-09074-4 · Cited by 51
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Shi, X., Wang, Z., Ren, W., Chen, L., Xu, C., Li, M., Fan, S., Xu, Y., Chen, M., Zheng, F., et al. (2022) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-00995-z · Cited by 45
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Shibata, S. B., Yoshimura, H., Ranum, P. T., Goodwin, A. T., & Smith, R. J. (2017) · Scientific Reports · doi:10.1038/s41598-017-09805-x · Cited by 44
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Qi, J., Huang, W., Lu, Y., Yang, X., Zhou, Y., Chen, T., Wang, X., Yu, Y., Sun, J., & Chai, R. (2023) · Neuroscience Bulletin · doi:10.1007/s12264-023-01130-w · Cited by 47
Iyer, A. A., & Groves, A. K. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2021.660748 · Cited by 40
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Self-citationWang, L., Xiao, R., Andrés‐Mateos, E., & Vandenberghe, L. H. (2017) · PLoS ONE · doi:10.1371/journal.pone.0182473 · Cited by 42
Self-citationOptical stimulation of neural tissue
Richardson, R. T., Ibbotson, M. R., Thompson, A. C., Wise, A. K., & Fallon, J. B. (2020) · Healthcare Technology Letters · doi:10.1049/htl.2019.0114 · Cited by 40
AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives
Zhang, L., Tan, F., Qi, J., Lu, Y., Wang, X., Yang, X., Chen, X., Zhang, X., Fan, J., Zhou, Y., et al. (2024) · Advanced Science · doi:10.1002/advs.202402166 · Cited by 40
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Cochlear histopathology in human genetic hearing loss: State of the science and future prospects
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Approaches and Vectors for Efficient Cochlear Gene Transfer in Adult Mouse Models
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Metadata
- 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
- Venue
- Nature Biotechnology
- Published
- 2017-02-06
- Volume
- 35
- Issue
- 3
- Pages
- 280-284
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- 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
- 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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