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Systemic AAV vectors for widespread and targeted gene delivery in rodents
Open accessAbstract
We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and noninvasive gene transfer to the central and peripheral nervous systems. However, a detailed protocol for generating and systemically delivering novel AAV variants was not previously available. In this protocol, we describe how to produce and intravenously administer AAVs to adult mice to specifically label and/or genetically manipulate cells in the nervous system and organs, including the heart. The procedure comprises three separate stages: AAV production, intravenous delivery, and evaluation of transgene expression. The protocol spans 8 d, excluding the time required to assess gene expression, and can be readily adopted by researchers with basic molecular biology, cell culture, and animal work experience. We provide guidelines for experimental design and choice of the capsid, cargo, and viral dose appropriate for the experimental aims. The procedures outlined here are adaptable to diverse biomedical applications, from anatomical and functional mapping to gene expression, silencing, and editing. Having developed AAV capsids that target sites throughout the body, here the authors describe how to produce and systemically administer these AAVs to rodents to label and/or genetically manipulate cells in the nervous system and visceral organs.
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In vivo genome editing using Staphylococcus aureus Cas9
Ran, F. A., Cong, L., Yan, W. X., Scott, D. A., Gootenberg, J. S., Kriz, A. J., Zetsche, B., Shalem, O., Wu, X., Makarova, K. S., et al. (2015) · Nature · doi:10.1038/nature14299 · Cited by 2,687
Chan, K. Y., Jang, M. J., Yoo, B. B., Greenbaum, A., Ravi, N., Wu, W.-L., Sánchez-Guardado, L., Lois, C., Mazmanian, S. K., Deverman, B. E., et al. (2017) · Nature Neuroscience · doi:10.1038/nn.4593 · Cited by 1,578
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Deverman, B. E., Pravdo, P. L., Simpson, B. P., Kumar, S. R., Chan, K. Y., Banerjee, A., Wu, W.-L., Yang, B., Huber, N., Pasca, S. P., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3440 · Cited by 1,061
Self-citationImmune responses to AAV vectors: overcoming barriers to successful gene therapy
Mingozzi, F., & High, K. A. (2013) · Blood · doi:10.1182/blood-2013-01-306647 · Cited by 955
Intact-brain analyses reveal distinct information carried by SNc dopamine subcircuits
Lerner, T. N., Shilyansky, C., Davidson, T. J., Evans, K. E., Beier, K. T., Zalocusky, K. A., Crow, A. K., Malenka, R. C., Luo, L., Tomer, R., et al. (2015) · Cell · doi:10.1016/j.cell.2015.07.014 · Cited by 794
Engineering adeno-associated viruses for clinical gene therapy
Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761
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Williams, E. K., Chang, R. B., Strochlic, D. E., Umans, B. D., Lowell, B. B., & Liberles, S. D. (2016) · Cell · doi:10.1016/j.cell.2016.05.011 · Cited by 645
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Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3469 · Cited by 556
Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501
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Hordeaux, J., Wang, Q., Katz, N., Buza, E. L., Bell, P., & Wilson, J. M. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.01.018 · Cited by 426
Jovičić, A., Roshan, R., Moisoi, N., Pradervand, S., Moser, R., Pillai, B., & Luthi-Carter, R. (2013) · The Journal of Neuroscience · doi:10.1523/jneurosci.0600-12.2013 · Cited by 251
Choi, J.-H., Yu, N.-K., Baek, G.-C., Bakes, J., Seo, D., Nam, H. J., Baek, S. H., Lim, C.-S., Lee, Y.-S., & Kaang, B.-K. (2014) · Molecular Brain · doi:10.1186/1756-6606-7-17 · Cited by 188
Xu, R., Janson, C., Mastakov, M., Lawlor, P., Young, D., Mouravlev, A., Fitzsimons, H., Choi, K.-L., Ma, H., Dragunow, M., et al. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301529 · Cited by 174
Viral Strategies for Targeting the Central and Peripheral Nervous Systems
Bedbrook, C. N., Deverman, B. E., & Gradinaru, V. (2018) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-080317-062048 · Cited by 168
Self-citationGao, K., Li, M., Zhong, L., Su, Q., Li, J., Li, S., He, R., Zhang, Y., Hendricks, G., Wang, J., et al. (2014) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2013.9 · Cited by 165
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Chamberlain, K., Riyad, J. M., & Weber, T. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.140 · Cited by 141
Transient transfection methods for clinical adeno-associated viral vector production
Wright, J. F. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.064 · Cited by 131
rAAV-compatible MiniPromoters for restricted expression in the brain and eye
de Leeuw, C. N., Korecki, A. J., Berry, G. E., Hickmott, J. W., Lam, S. L., Lengyell, T. C., Bonaguro, R. J., Borretta, L. J., Chopra, V., Chou, A. Y., et al. (2016) · Molecular Brain · doi:10.1186/s13041-016-0232-4 · Cited by 76
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Metadata
- Authors
- Challis, R. C., Ravindra Kumar, S., Chan, K. Y., Challis, C., Beadle, K., Jang, M. J., Kim, H. M., Rajendran, P. S., Tompkins, J. D., Shivkumar, K., Deverman, B. E., & Gradinaru, V.
- Venue
- Nature Protocols
- Published
- 2019-01-09
- Volume
- 14
- Issue
- 2
- Pages
- 379-414
- ISSN
- 1750-2799 (ISSN-L)
- 1754-2189
- OpenAlex
- W2910480910
- Cited by
- 409
- References
- 18
- FWCI
- 19.77
- Type
- journal article
- Language
- en
- Open access
- green
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