Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
Open accessAbstract
Adeno-associated virus (AAV) capsid libraries have generated improved transgene delivery vectors. We designed an AAV library construct, iTransduce, that combines a peptide library on the AAV9 capsid with a Cre cassette to enable sensitive detection of transgene expression. After only two selection rounds of the library delivered intravenously in transgenic mice carrying a Cre-inducible fluorescent protein, we flow sorted fluorescent cells from brain, and DNA sequencing revealed two dominant capsids. One of the capsids, termed AAV-F, mediated transgene expression in the brain cortex more than 65-fold (astrocytes) and 171-fold (neurons) higher than the parental AAV9. High transduction efficiency was sex-independent and sustained in two mouse strains (C57BL/6 and BALB/c), making it a highly useful capsid for CNS transduction of mice. Future work in large animal models will test the translation potential of AAV-F.
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AAV-F achieved 65-fold higher astrocyte transduction and 171-fold higher neuron transduction than parental AAV9.
“with 65-fold and 171-fold higher transduction of astrocytes and neurons than parental AAV9 respectively [129].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [129]
AAV-F mediated successful CNS transduction in C57BL/6J and BALB/c mice.
“the iTransduce capsid-engineering platform yielded a new variant, namely AAV-F, which mediated successful CNS transduction in C57BL/6 J and BALB/c mice”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [129]
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381 · see citing works
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163 · see citing works
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
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 · see citing works
Hinderer, C., Katz, N., Buza, E. L., Dyer, C., Goode, T., Bell, P., Richman, L. K., & Wilson, J. M. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.015 · Cited by 814 · see citing works
Generation of transgenic non-human primates with germline transmission
Sasaki, E., Suemizu, H., Shimada, A., Hanazawa, K., Oiwa, R., Kamioka, M., Tomioka, I., Sotomaru, Y., Hirakawa, R., Eto, T., et al. (2009) · Nature · doi:10.1038/nature08090 · Cited by 712 · see citing works
Aurnhammer, C., Haase, M., Müther, N., Mück-Häusl, M., Rauschhuber, C., Huber, I., Nitschko, H., Busch, U., Sing, A., Ehrhardt, A., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2011.034 · Cited by 495 · see citing works
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
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 · see citing works
Microvesicle-associated AAV Vector as a Novel Gene Delivery System
Maguire, C. A., Balaj, L., Sivaraman, S., Crommentuijn, M. H., Ericsson, M., Mincheva-Nilsson, L., Baranov, V., Gianni, D., Tannous, B. A., Sena-Esteves, M., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2011.303 · Cited by 283 · see citing works
Self-citationThe GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier
Hordeaux, J., Yuan, Y., Clark, P. M., Wang, Q., Martino, R. A., Sims, J. J., Bell, P., Raymond, A., Stanford, W. L., & Wilson, J. M. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.013 · Cited by 217 · see citing works
Tse, L. V., Klinc, K. A., Madigan, V. J., Castellanos Rivera, R. M., Wells, L. F., Havlik, L. P., Smith, J. K., Agbandje-McKenna, M., & Asokan, A. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1704766114 · Cited by 210 · see citing works
Gray, S. J., Blake, B. L., Criswell, H. E., Nicolson, S. C., Samulski, R. J., & McCown, T. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.292 · Cited by 185 · see citing works
György, B., Meijer, E. J., Ivanchenko, M. V., Tenneson, K., Émond, F., Hanlon, K. S., Indzhykulian, A. A., Volak, A., Karavitaki, K. D., Tamvakologos, P. I., et al. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.11.003 · Cited by 162 · see citing works
Self-citationPaulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.021 · Cited by 161 · see citing works
Exosome-associated AAV vector as a robust and convenient neuroscience tool
Hudry, E., Martin, C., Gandhi, S., György, B., Scheffer, D. I., Mu, D., Merkel, S. F., Mingozzi, F., Fitzpatrick, Z., Dimant, H., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.11 · Cited by 146 · see citing works
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Park, J. E., Zhang, X. F., Choi, S., Okahara, J., Sasaki, E., & Silva, A. C. (2016) · Scientific Reports · doi:10.1038/srep34931 · Cited by 95 · see citing works
D'Costa, S., Blouin, V., Broucque, F., Penaud-Budloo, M., Fçranois, A., Perez, I. C., Le Bec, C., Moullier, P., Snyder, R. O., & Ayuso, E. (2016) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2016.19 · Cited by 89 · see citing works
Körbelin, J., Sieber, T., Michelfelder, S., Lunding, L. P., Spies, E., Hunger, A., Alawi, M., Rapti, K., Indenbirken, D., Müller, O. J., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.62 · Cited by 88 · see citing works
Koerber, J. T., Maheshri, N., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Protocols · doi:10.1038/nprot.2006.93 · Cited by 86 · see citing works
Cellular transduction mechanisms of adeno-associated viral vectors
Berry, G. E., & Asokan, A. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.08.001 · Cited by 78 · see citing works
Dashkoff, J., Lerner, P., Truong, N., Klickstein, J. A., Fan, Z., Mu, D., Maguire, C. A., Hyman, B. T., & Hudry, E. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.81 · Cited by 56 · see citing works
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Matsuzaki, Y., Tanaka, M., Hakoda, S., Masuda, T., Miyata, R., Konno, A., & Hirai, H. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.016 · Cited by 48 · see citing works
Tropism-modified AAV Vectors Overcome Barriers to Successful Cutaneous Therapy
Sallach, J., Pasquale, G. D., Larcher, F., Niehoff, N., Rübsam, M., Huber, A., Chiorini, J., Almarza, D., Eming, S. A., Ulus, H., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.14 · Cited by 47 · see citing works
High Capsid–Genome Correlation Facilitates Creation of AAV Libraries for Directed Evolution
Nonnenmacher, M. E., van Bakel, H., Hajjar, R. J., & Weber, T. (2015) · Molecular Therapy · doi:10.1038/mt.2015.3 · Cited by 32 · see citing works
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Igarashi, H., Koizumi, K., Kaneko, R., Ikeda, K., Egawa, R., Yanagawa, Y., Muramatsu, S., Onimaru, H., Ishizuka, T., & Yawo, H. (2016) · PLoS ONE · doi:10.1371/journal.pone.0155687 · Cited by 27 · see citing works
Watters, A. K., Rom, S., Hill, J. D., Dematatis, M. K., Zhou, Y., Merkel, S. F., Andrews, A. M., Cenna, J. M., Potula, R., Skuba, A., et al. (2015) · Stem Cells and Development · doi:10.1089/scd.2014.0497 · Cited by 25 · see citing works
Self-citationBioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle
Paulk, N. K., Pekrun, K., Charville, G. W., Maguire, K., Wosczyna, M. N., Xu, J., Zhang, Y., Lisowski, L., Yoo, B., Vilches-Moure, J. G., et al. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.06.001 · Cited by 19 · see citing works
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Bulcha, J. T., Wang, Y., Ma, H., Tai, P. W. L., & Gao, G. (2021) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-021-00487-6 · Cited by 1,404 · see citing works
Strategies for delivering therapeutics across the blood–brain barrier
Terstappen, G. C., Meyer, A. H., Bell, R. D., & Zhang, W. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-021-00139-y · Cited by 1,138 · see citing works
An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery
Huang, Q., Chan, K. Y., Wu, J. H., Botticello-Romero, N. R., Zheng, Q., Lou, S., Keyes, C., Svanbergsson, A., Johnston, J., Mills, A., et al. (2024) · Science · doi:10.1126/science.adm8386 · Cited by 200 · see citing works
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning
Nonnenmacher, M., Wang, W., Child, M. A., Ren, X.-Q., Huang, C., Ren, A. Z., Tocci, J., Chen, Q., Bittner, K., Tyson, K., et al. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.12.006 · Cited by 168 · see citing works
Adeno-Associated Virus Toolkit to Target Diverse Brain Cells
Challis, R. C., Ravindra Kumar, S., Chen, X., Goertsen, D., Coughlin, G. M., Hori, A. M., Chuapoco, M. R., Otis, T. S., Miles, T. F., & Gradinaru, V. (2022) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-111020-100834 · Cited by 148 · see citing works
Chuapoco, M. R., Flytzanis, N. C., Goeden, N., Octeau, J. C., Roxas, K. M., Chan, K. Y., Scherrer, J., Winchester, J., Blackburn, R. J., Campos, L., et al. (2023) · Nature Nanotechnology · doi:10.1038/s41565-023-01419-x · Cited by 132 · see citing works
Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems
Chen, X., Kumar, S. R., Adams, C. D., Yang, D., Wang, T., Wolfe, D. A., Arokiaraj, C. M., Ngo, V., Campos, L., Griffiths, J. A., et al. (2022) · Neuron · doi:10.1016/j.neuron.2022.05.003 · Cited by 131 · see citing works
Griciuc, A., Federico, A. N., Natasan, J.-S., Forte, A. M., McGinty, D., Nguyen, H., Volak, A., LeRoy, S. G., Gandhi, S., Lerner, P., et al. (2020) · Human Molecular Genetics · doi:10.1093/hmg/ddaa179 · Cited by 125 · see citing works
Self-citationYao, Y., Wang, J., Liu, Y., Qu, Y., Wang, K., Zhang, Y., Chang, Y., Yang, Z., Wan, J., Liu, J., et al. (2022) · Nature Biomedical Engineering · doi:10.1038/s41551-022-00938-7 · Cited by 120 · see citing works
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Krolak, T., Chan, K. Y., Kaplan, L., Huang, Q., Wu, J. H., Zheng, Q., Kozareva, V., Beddow, T., Tobey, I. G., Pacouret, S., et al. (2022) · Nature Cardiovascular Research · doi:10.1038/s44161-022-00046-4 · Cited by 119 · see citing works
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Ntetsika, T., Papathoma, P.-E., & Markaki, I. (2021) · Molecular Medicine · doi:10.1186/s10020-021-00279-2 · Cited by 112 · see citing works
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Suárez-Amarán, L., Song, L., Tretiakova, A. P., Mikhail, S., & Samulski, R. J. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.064 · Cited by 87 · see citing works
Jensen, T. L., Gøtzsche, C. R., & Woldbye, D. P. D. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2021.695937 · Cited by 85 · see citing works
Chen, W., Yao, S., Wan, J., Tian, Y., Huang, L., Wang, S., Akter, F., Wu, Y., Yao, Y., & Zhang, X. (2021) · Journal of Controlled Release · doi:10.1016/j.jconrel.2021.03.029 · Cited by 81 · see citing works
Becker, J., Fakhiri, J., & Grimm, D. (2022) · Pathogens · doi:10.3390/pathogens11070756 · Cited by 80 · see citing works
Huang, Q., Chen, A. T., Chan, K. Y., Sorensen, H., Barry, A. J., Azari, B., Zheng, Q., Beddow, T., Zhao, B., Tobey, I. G., et al. (2023) · PLoS Biology · doi:10.1371/journal.pbio.3002112 · Cited by 80 · see citing works
Mechanisms of receptor-mediated transcytosis at the blood-brain barrier
Baghirov, H. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.113595 · Cited by 81 · see citing works
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Eid, F.-E., Chen, A. T., Chan, K. Y., Huang, Q., Zheng, Q., Tobey, I. G., Pacouret, S., Brauer, P. P., Keyes, C., Powell, M. M., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-50555-y · Cited by 74 · see citing works
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Ivanchenko, M. V., Hanlon, K. S., Hathaway, D. M., Klein, A. J., Peters, C. W., Li, Y., Tamvakologos, P. I., Nammour, J., Maguire, C. A., & Corey, D. P. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.03.019 · Cited by 68 · see citing works
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Ghauri, M. S., & Ou, L. (2023) · Biology · doi:10.3390/biology12020186 · Cited by 61 · see citing works
López-Gordo, E., Chamberlain, K., Riyad, J. M., Kohlbrenner, E., & Weber, T. (2024) · Viruses · doi:10.3390/v16030442 · Cited by 61 · see citing works
Current and Emerging Strategies for Enhancing Antibody Delivery to the Brain
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Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
Large, E. E., Silveria, M., Zane, G. M., Weerakoon, O., & Chapman, M. S. (2021) · Viruses · doi:10.3390/v13071336 · Cited by 54 · see citing works
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Jang, M. J., Coughlin, G. M., Jackson, C. R., Chen, X., Chuapoco, M. R., Vendemiatti, J., Wang, A. Z., & Gradinaru, V. (2023) · Nature Biotechnology · doi:10.1038/s41587-022-01648-w · Cited by 54 · see citing works
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Liu, Q. L., Wu, Y., Wang, H., Jia, F., & Xu, F. (2022) · Neuroscience Bulletin · doi:10.1007/s12264-022-00949-z · Cited by 51 · see citing works
Kolesnik, V. V., Nurtdinov, R. F., Oloruntimehin, E. S., Karabelsky, A. V., & Malogolovkin, A. S. (2024) · Clinical and Translational Medicine · doi:10.1002/ctm2.1607 · Cited by 51 · see citing works
Alves, C. R. R., Ha, L. L., Yaworski, R., Sutton, E. R., Lazzarotto, C. R., Christie, K. A., Reilly, A., Beauvais, A., Doll, R. M., de la Cruz, D., et al. (2023) · Nature Biomedical Engineering · doi:10.1038/s41551-023-01132-z · Cited by 75 · see citing works
Self-citationHuang, L., Wan, J., Wu, Y., Tian, Y., Yao, Y., Yao, S., Ji, X., Wang, S., Su, Z., & Xu, H. (2021) · Life Sciences · doi:10.1016/j.lfs.2021.119142 · Cited by 47 · see citing works
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 · see citing works
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Self-citationLin, K., Zhong, X., Li, L., Ying, M., Yang, T., Zhang, Z., He, X., & Xu, F. (2020) · Molecular Brain · doi:10.1186/s13041-020-00679-1 · Cited by 45 · see citing works
Hana, S., Peterson, M. A., McLaughlin, H., Marshall, E., Fabian, A. J., McKissick, O. P., Koszka, K., Marsh, G., Craft, M., Xu, S., et al. (2021) · Gene Therapy · doi:10.1038/s41434-021-00224-2 · Cited by 43 · see citing works
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Peters, C. W., Hanlon, K. S., Ivanchenko, M. V., Zinn, E. M., Linarte, E., Li, Y., Levy, J. M., Liu, D. R., Kleinstiver, B. P., Indzhykulian, A. A., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.06.007 · Cited by 36 · see citing works
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Ertl, H. C. J. (2023) · Drugs · doi:10.1007/s40265-023-01836-1 · Cited by 39 · see citing works
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Słyk, Ż., Stachowiak, N., & Małecki, M. (2024) · Biomedicines · doi:10.3390/biomedicines12071523 · Cited by 39 · see citing works
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Zhou, X., Liu, J., Xiao, S., Liang, X., Li, Y., Mo, F., Xin, X., Yang, Y., & Gao, C. (2024) · International Journal of Nanomedicine · doi:10.2147/ijn.s459905 · Cited by 33 · see citing works
Meng, Y., Sun, D., Qin, Y., Dong, X., Luo, G., & Liu, Y. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.019 · Cited by 29 · see citing works
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DeRosa, S., Salani, M., Smith, S., Sangster, M., Miller-Browne, V., Wassmer, S., Xiao, R., Vandenberghe, L. H., Slaugenhaupt, S. A., Misko, A. L., et al. (2021) · Human Molecular Genetics · doi:10.1093/hmg/ddab093 · Cited by 20 · see citing works
Base editing as a genetic treatment for spinal muscular atrophyPreprint
Alves, C. R. R., Ha, L. L., Yaworski, R., Lazzarotto, C. R., Christie, K. A., Reilly, A., Beauvais, A., Doll, R. M., de la Cruz, D., Maguire, C. A., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.01.20.524978 · Cited by 20 · see citing works
Self-citationNext-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
Björklund, T., & Davidsson, M. (2021) · Journal of Parkinson s Disease · doi:10.3233/jpd-212674 · Cited by 19 · see citing works
Glioblastoma Multiforme Selective Nanomedicines for Improved Anti-Cancer Treatments
Duskey, J. T., Rinaldi, A., Ottonelli, I., Caraffi, R., Benedictis, C. A. D., Sauer, A. K., Tosi, G., Vandelli, M. A., Ruozi, B., & Grabrucker, A. M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14071450 · Cited by 19 · see citing works
Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases
Li, L., Vasan, L., Kartono, B., Clifford, K. P., Attarpour, A., Sharma, R., Mandrozos, M., Kim, A., Zhao, W., Belotserkovsky, A., et al. (2023) · Biomedicines · doi:10.3390/biomedicines11102725 · Cited by 19 · see citing works
Shi, C., Tian, L., Zheng, W., Zhu, Y., Sun, P., Liu, L., Liu, W., Song, Y., Xia, X., Xue, X., et al. (2022) · Emerging Microbes & Infections · doi:10.1080/22221751.2022.2078226 · Cited by 18 · see citing works
Non-viral approaches for gene therapy and therapeutic genome editing across the blood–brain barrier
Xie, R., Wang, Y., Burger, J. C., Li, D., Zhu, M., & Gong, S. (2023) · Med-X · doi:10.1007/s44258-023-00004-0 · Cited by 20 · see citing works
Kawabata, H., Konno, A., Matsuzaki, Y., Sato, Y., Kawachi, M., Aoki, R., Tsutsumi, S., Togai, S., Kobayashi, R., Horii, T., et al. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101185 · Cited by 17 · see citing works
Majumdar, M., & Badwaik, H. R. (2024) · Current Drug Targets · doi:10.2174/0113894501312703240826070530 · Cited by 16 · see citing works
AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery
Jackson, C. B., Richard, A. S., Ojha, A., Conkright, K. A., Trimarchi, J. M., Bailey, C. C., Alpert, M. D., Kay, M. A., Farzan, M., & Choe, H. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.11.004 · Cited by 16 · see citing works
Macdonald, J., Marx, J., & Büning, H. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.169 · Cited by 16 · see citing works
Distributional comparison of different AAV vectors after unilateral cochlear administration
Han, S., Xu, Z., Wang, S., Tang, H., Hu, S., Wang, H., Guan, G., & Shu, Y. (2023) · Gene Therapy · doi:10.1038/s41434-023-00431-z · Cited by 15 · see citing works
Therapeutic Application and Structural Features of Adeno-Associated Virus Vector
Matsuzaka, Y., & Yashiro, R. (2024) · Current Issues in Molecular Biology · doi:10.3390/cimb46080499 · Cited by 15 · see citing works
Matsuzaki, Y., Fukai, Y., Konno, A., & Hirai, H. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101337 · Cited by 15 · see citing works
Fakhiri, J., Landegger, L. D., & Grimm, D. (2020) · Hearing Research · doi:10.1016/j.heares.2020.108092 · Cited by 15 · see citing works
rAAV expressing recombinant neutralizing antibody for the botulinum neurotoxin type A prophylaxis
Derkaev, A. A., Ryabova, E. I., Esmagambetov, I. B., Shcheblyakov, D. V., Godakova, S. A., Vinogradova, I. D., Noskov, A. N., Logunov, D. Y., Naroditsky, B. S., & Al, G. (2022) · Frontiers in Microbiology · doi:10.3389/fmicb.2022.960937 · Cited by 15 · see citing works
rAAV expressing recombinant antibody for emergency prevention and long-term prophylaxis of COVID-19
Esmagambetov, I. B., Ryabova, E. I., Derkaev, A. A., Shcheblyakov, D. V., Dolzhikova, I. V., Favorskaya, I. A., Grousova, D. M., Dovgiy, M. A., Prokofiev, V. V., Gosudarev, A. I., et al. (2023) · Frontiers in Immunology · doi:10.3389/fimmu.2023.1129245 · Cited by 14 · see citing works
Smith, E. J., Farshim, P. P., Flomen, R. H., Jones, S. T., McAteer, S. J., Deverman, B. E., Gradinaru, V., & Bates, G. P. (2021) · Brain Communications · doi:10.1093/braincomms/fcab105 · Cited by 14 · see citing works
Santoscoy, M. C., Espinoza, P., de la Cruz, D., Mahamdeh, M., Starr, J. R., Patel, N. J., & Maguire, C. A. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.05.001 · Cited by 13 · see citing works
Self-citationHanlon, K. S., Cheng, M. L., Ferrer, R. M., Ryu, J. R., Lee, B., de la Cruz, D., Patel, N. J., Espinoza, P., Santoscoy, M. C., Gong, Y., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.05.040 · Cited by 14 · see citing works
Self-citationLuo, N., Lin, K., Cai, Y., Zhang, Z., Sui, X., Han, Z., Yuan, W., Xing, J., Gui, Z., Liu, G., et al. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.01.021 · Cited by 13 · see citing works
Wong, P. K., Cheah, F., Syafruddin, S. E., Mohtar, M. A., Azmi, N., Ng, P. Y., & Chua, E. W. (2021) · Frontiers in Pediatrics · doi:10.3389/fped.2021.592571 · Cited by 14 · see citing works
In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges
Cantore, A., Fraldi, A., Meneghini, V., & Gritti, A. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2021.774618 · Cited by 13 · see citing works
Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders
Wang, J., Gao, G., & Wang, D. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00391 · Cited by 10 · see citing works
Martino, R. A., Wang, Q., Xu, H. Y., Hu, G. B., Bell, P. L., Arroyo, E. J., Sims, J. J., & Wilson, J. M. (2023) · Journal of Virology · doi:10.1128/jvi.00174-23 · Cited by 12 · see citing works
Li, M., Zhong, Y., Zhu, M., Pang, C., Xiao, L. X., Bu, Y., Li, H., Diao, Y., Yang, C. J., & Liu, D. (2024) · The Analyst · doi:10.1039/d4an00404c · Cited by 10 · see citing works
Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice
Alves, C. R. R., Das, S., Krishnan, V., Ha, L. L., Fox, L. R., Stutzman, H. E., Shamber, C. E., Kalailingam, P., McCarthy, S., Cardenas, C. L. L., et al. (2025) · Nature Biomedical Engineering · doi:10.1038/s41551-025-01499-1 · Cited by 13 · see citing works
Self-citationTissue blood volumes and weights in adult beagle dogs.
Cuddihy, R. G., Hall, R. P., HOBBS, C. H., Boecker, B. B., & Muggenburg, B. A. (1972) · OSTI OAI (U.S. Department of Energy Office of Scientific and Technical Information) · Cited by 10 · see citing works
Systematic multi-trait AAV capsid engineering for efficient gene deliveryPreprint
Eid, F.-E., Chen, A. T., Chan, K. Y., Huang, Q., Zheng, Q., Tobey, I. G., Pacouret, S., Brauer, P. P., Keyes, C., Powell, M. M., et al. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.12.22.521680 · Cited by 10 · see citing works
Chadderton, N., Palfi, A., Maloney, D. M., Carrigan, M., Finnegan, L. K., Hanlon, K. S., Shortall, C., O’Reilly, M., Humphries, P., Cassidy, L., et al. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15020322 · Cited by 9 · see citing works
Self-citationPowell, S., & McCown, T. J. (2024) · Journal of Virology · doi:10.1128/jvi.01681-24 · Cited by 10 · see citing works
Nieland, L., Vrijmoet, A. B., Jetten, I. W., Rufino‐Ramos, D., de Reus, A. J. E. M., Breyne, K., Kleinstiver, B. P., Maguire, C. A., Broekman, M. L. D., Breakefield, X. O., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.11.023 · Cited by 9 · see citing works
Self-citationNext-generation programmable cell therapies for precision medicine
Teixeira, A. P., Aubel, D., & Fussenegger, M. (2026) · Nature Reviews Genetics · doi:10.1038/s41576-026-00945-3 · Cited by 11 · see citing works
Optogenetic activation of the diaphragm
Benevides, E. S., Sunshine, M. D., Rana, S. D., & Fuller, D. D. (2022) · Scientific Reports · doi:10.1038/s41598-022-10240-w · Cited by 9 · see citing works
Focused ultrasound‐mediated <i>APOE4</i> knockdown in mouse brain
Zheng, K., Tsitsos, F. N., Batts, A. J., Ji, R., Nuriel, T., Konofagou, E. E., & Leong, K. W. (2025) · Alzheimer's & Dementia · doi:10.1002/alz.70464 · Cited by 8 · see citing works
Karan, K. R., Andrzejewski, S., Stiles, K. M., Hackett, N. R., & Crystal, R. G. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.112 · Cited by 8 · see citing works
Targeting AAV vectors to the CNS via de novo engineered capsid-receptor interactionsPreprint
Huang, Q., Chen, A. T., Chan, K. Y., Sorensen, H., Barry, A. J., Azari, B., Beddow, T., Zheng, Q., Zhao, B., Tobey, I. G., et al. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.10.31.514553 · Cited by 8 · see citing works
Production of Spinocerebellar Ataxia Type 3 Model Mice by Intravenous Injection of AAV-PHP.B Vectors
Konno, A., Shinohara, Y., & Hirai, H. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25137205 · Cited by 7 · see citing works
Intratumoral delivery of 4-1BBL boosts IL-12-triggered anti-glioma immunity
Lunavat, T. R., Nieland, L., van de Looij, S. M., de Reus, A. J. E. M., Couturier, C., Farran, C. A. E., Miller, T. E., Lill, J. K., Schübel, M., Xiao, T., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.08.028 · Cited by 7 · see citing works
Cwerman‐Thibault, H., Malko-Baverel, V., Guilloux, G. L., Ratcliffe, E., Mouri, D., Torres-Cuevas, I., Millán, I., Saubaméa, B., Mignon, V., Boespflug‐Tanguy, O., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.05.030 · Cited by 7 · see citing works
Weinmann, J., Söllner, J. F., Abele, S., Zimmermann, G., Zuckschwerdt, K., Mayer, C. A., Danner-Liskus, J., Peltzer, A., Schuler, M. J., Lamla, T., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.109 · Cited by 7 · see citing works
Cappella, M., Elouej, S., & Biferi, M. G. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.662837 · Cited by 7 · see citing works
Duarte, F. T., Ramosaj, M., Hasanovic, E., Regio, S., Sipion, M., Rey, M. J., & Déglon, N. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.03.013 · Cited by 7 · see citing works
Boosters for adeno-associated virus (AAV) vector (r)evolution
Szumska, J., & Grimm, D. (2022) · Cytotherapy · doi:10.1016/j.jcyt.2022.07.005 · Cited by 6 · see citing works
- Authors
- Hanlon, K. S., Meltzer, J. C., Buzhdygan, T., Cheng, M. J., Sena-Esteves, M., Bennett, R. E., Sullivan, T. P., Razmpour, R., Gong, Y., Ng, C., Nammour, J., Maiz, D., Dujardin, S., Ramirez, S. H., Hudry, E., & Maguire, C. A.
- Affiliations
- Harvard University (US)
- Temple University (US)
- Corresponding author
- Maguire, Casey A.
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2019-12
- Volume
- 15
- Pages
- 320-332
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC6881693
- OpenAlex
- W2982053609
- Cited by
- 148
- References
- 26
- Citations by year
- 2026: 15 · 2025: 21 · 2024: 32 · 2023: 23 · 2022: 23 · 2021: 26 · 2020: 7
- FWCI
- 8.44
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- eScholarship@UMassChan (submittedVersion, other-oa)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- National Institutes of Health
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- adeno-associated virus, AAV9, transgene expression, peptide libraries, intravenous delivery, flow cytometric cell sorting, astrocytes, neurons, mouse strain comparison
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