The DOI matches its Crossref record; 37 of the 37 references it cites were found in a registry or an index.
Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c
Open accessAbstract
Because there are currently no biological treatments for hearing loss, we sought to advance gene therapy approaches to treat genetic deafness. We focused on Usher syndrome, a devastating genetic disorder that causes blindness, balance disorders and profound deafness, and studied a knock-in mouse model, Ush1c c.216G>A, for Usher syndrome type IC (USH1C). As restoration of complex auditory and balance function is likely to require gene delivery systems that target auditory and vestibular sensory cells with high efficiency, we delivered wild-type Ush1c into the inner ear of Ush1c c.216G>A mice using a synthetic adeno-associated viral vector, Anc80L65, shown to transduce 80–90% of sensory hair cells. We demonstrate recovery of gene and protein expression, restoration of sensory cell function, rescue of complex auditory function and recovery of hearing and balance behavior to near wild-type levels. The data represent unprecedented recovery of inner ear function and suggest that biological therapies to treat deafness may be suitable for translation to humans with genetic inner ear disorders.
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Cites
Defective myosin VIIA gene responsible for Usher syndrome type IB
Well, D., Blanchard, S., Kaplan, J., Guilford, P., Gibson, F., Walsh, J., Mburu, P., Varela, A., Levilliers, J., Weston, M. D., et al. (1995) · Nature · doi:10.1038/374060a0 · Cited by 1,068
Bork, J. M., Peters, L. M., Riazuddin, S., Bernstein, S. L., Ahmed, Z. M., Ness, S. L., Polomeno, R., Ramesh, A., Schloss, M., Srisailpathy, C. R. S., et al. (2001) · The American Journal of Human Genetics · doi:10.1086/316954 · Cited by 555
Lighting up the Senses: FM1-43 Loading of Sensory Cells through Nonselective Ion Channels
Meyers, J. R., MacDonald, R. B., Duggan, A., Lenzi, D., Standaert, D. G., Corwin, J. T., & Corey, D. P. (2003) · The Journal of Neuroscience · doi:10.1523/jneurosci.23-10-04054.2003 · Cited by 526
Duan, D., Sharma, P., Yang, J., Yue, Y., Dudus, L., Zhang, Y., Fisher, K. J., & Engelhardt, J. F. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8568-8577.1998 · Cited by 522
Mutation of CDH23, encoding a new member of the cadherin gene family, causes Usher syndrome type 1D
Bolz, H., von Brederlow, B., Ramírez, A., Bryda, E. C., Kutsche, K., Nothwang, H. G., Seeliger, M., Cabrera, M. D. C.-S., Vila, M. C., Molina, O. P., et al. (2001) · Nature Genetics · doi:10.1038/83667 · Cited by 497
Noben-Trauth, K., Zheng, Q. Y., & Johnson, K. R. (2003) · Nature Genetics · doi:10.1038/ng1226 · Cited by 479
Verpy, E., Leibovici, M., Zwaenepoel, I., Liu, X.-Z., Gal, A., Salem, N., Mansour, A., Blanchard, S., Kobayashi, I., Keats, B. J. B., et al. (2000) · Nature Genetics · doi:10.1038/79171 · Cited by 471
Boeda, B. (2002) · The EMBO Journal · doi:10.1093/emboj/cdf689 · Cited by 447
FM1-43 Dye Behaves as a Permeant Blocker of the Hair-Cell Mechanotransducer Channel
Gale, J. E., Marcotti, W., Kennedy, H. J., Kros, C. J., & Richardson, G. P. (2001) · The Journal of Neuroscience · doi:10.1523/jneurosci.21-18-07013.2001 · Cited by 442
Mutations of the Protocadherin Gene PCDH15 Cause Usher Syndrome Type 1F
Ahmed, Z. M., Riazuddin, S., Bernstein, S. L., Ahmed, Z., Khan, S., Griffith, A. J., Morell, R. J., Friedman, T. B., Riazuddin, S., & Wilcox, E. R. (2001) · The American Journal of Human Genetics · doi:10.1086/321277 · Cited by 415
Cited by
Engineering adeno-associated virus vectors for gene therapy
Li, C., & Samulski, R. J. (2020) · Nature Reviews Genetics · doi:10.1038/s41576-019-0205-4 · Cited by 1,175
Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents
Gao, X., Tao, Y., Lamas, V., Huang, M., Yeh, W.-H., Pan, B., Hu, Y.-J., Hu, J. H., Thompson, D. B., Shu, Y., et al. (2018) · Nature · doi:10.1038/nature25164 · Cited by 536
Self-citationTherapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.02.017 · Cited by 369
Self-citationDual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model
Akil, O., Dyka, F., Calvet, C., Emptoz, A., Lahlou, G., Nouaille, S., Boutet de Monvel, J., Hardelin, J.-P., Hauswirth, W. W., Avan, P., et al. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1817537116 · Cited by 300
Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65
Hudry, E., Andres-Mateos, E., Lerner, E. P., Volak, A., Cohen, O., Hyman, B. T., Maguire, C. A., & Vandenberghe, L. H. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.07.006 · Cited by 84
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Metadata
- Authors
- Pan, B., Askew, C., Galvin, A., Heman-Ackah, S., Asai, Y., Indzhykulian, A. A., Jodelka, F. M., Hastings, M. L., Lentz, J. J., Vandenberghe, L. H., Holt, J. R., & Géléoc, G. S.
- Venue
- Nature Biotechnology
- Published
- 2017-02-06
- Volume
- 35
- Issue
- 3
- Pages
- 264-272
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- OpenAlex
- W2586573214
- Cited by
- 294
- References
- 37
- FWCI
- 21.53
- Type
- journal article
- Language
- en
- Open access
- green
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- Pan, B., Askew, C., Galvin, A., Heman-Ackah, S., Asai, Y., Indzhykulian, A. A., Jodelka, F. M., Hastings, M. L., Lentz, J. J., Vandenberghe, L. H., Holt, J. R., & Géléoc, G. S. (2017). Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c. Nature Biotechnology. https://doi.org/10.1038/nbt.3801
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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