Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning

Ultrafast and memory-efficient alignment of short DNA sequences to the human genome.

Langmead, B., Trapnell, C., Pop, M., & Salzberg, S. L. (2009) · Genome Biology · doi:10.1186/gb-2009-10-3-r25 · Cited by 21,849 · see citing works

Adeno-associated virus vector as a platform for gene therapy delivery

Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444 · see citing works

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

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

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 · see citing works

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490 · 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

In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

Dalkara, D., Byrne, L. C., Klimczak, R. R., Visel, M., Yin, L., Merigan, W. H., Flannery, J. G., & Schaffer, D. V. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005708 · Cited by 734 · see citing works

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696 · see citing works

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy

Xie, Q., Bu, W., Bhatia, S., Hare, J., Somasundaram, T., Azzi, A., & Chapman, M. S. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.162250899 · Cited by 612 · see citing works

Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

Maheshri, N., Koerber, J. T., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Biotechnology · doi:10.1038/nbt1182 · Cited by 538 · cited 1× · see citing works

The stringdist Package for Approximate String Matching

Mark, P. J. (2014) · The R Journal · doi:10.32614/rj-2014-011 · Cited by 477 · see citing works

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Lisowski, L., Dane, A. P., Chu, K., Zhang, Y., Cunningham, S. C., Wilson, E. M., Nygaard, S., Grompe, M., Alexander, I. E., & Kay, M. A. (2014) · Nature · doi:10.1038/nature12875 · Cited by 450 · 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

Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

Wang, Z., Ma, H.-I., Li, J., Sun, L., Zhang, J., & Xiao, X. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302133 · Cited by 413 · see citing works

Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors

Müller, O. J., Kaul, F., Weitzman, M. D., Pasqualini, R., Arap, W., Kleinschmidt, J. A., & Trepel, M. (2003) · Nature Biotechnology · doi:10.1038/nbt856 · Cited by 411 · see citing works

Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

Cearley, C. N., & Wolfe, J. H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.015 · Cited by 409 · see citing works

GFAP promoter elements required for region‐specific and astrocyte‐specific expression

Lee, Y., Messing, A., Su, M., & Brenner, M. (2008) · Glia · doi:10.1002/glia.20622 · Cited by 406 · see citing works

Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306 · see citing works

Comparative Analysis of Adeno-Associated Viral Vector Serotypes 1, 2, 5, 7, And 8 in Mouse Brain

Taymans, J.-M., Vandenberghe, L. H., Haute, C. V. D., Thiry, I., Deroose, C. M., Mortelmans, L., Wilson, J. M., Debyser, Z., & Baekelandt, V. (2007) · Human Gene Therapy · doi:10.1089/hum.2006.178 · Cited by 305 · see citing works

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291 · see citing works

Erratum: Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248 · see citing works

A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases

Körbelin, J., Dogbevia, G., Michelfelder, S., Ridder, D. A., Hunger, A., Wenzel, J., Seismann, H., Lampe, M., Bannach, J., Pasparakis, M., et al. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201506078 · Cited by 243 · see citing works

Starcode: sequence clustering based on all-pairs search

Zorita, E. V., Cuscó, P., & Filion, G. J. P. (2015) · Bioinformatics · doi:10.1093/bioinformatics/btv053 · Cited by 241 · see citing works

Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

Huang, Q., Chan, K. Y., Tobey, I. G., Chan, Y. A., Poterba, T., Boutros, C. L., Balazs, A. B., Daneman, R., Bloom, J. M., Seed, C., et al. (2019) · PLOS ONE · doi:10.1371/journal.pone.0225206 · Cited by 234 · see citing works

In vitro selection of viral vectors with modified tropism: the adeno-associated virus display

Perabò, L., Büning, H., Kofler, D. M., Ried, M. U., Girod, A., Wendtner, C., Enssle, J., & Hallek, M. J. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00123-0 · Cited by 232 · see citing works

Structural insight into the unique properties of adeno-associated virus serotype 9

DiMattia, M. A., Nam, H.-J., Van Vliet, K., Mitchell, M., Bennett, A., Gurda, B. L., McKenna, R., Olson, N. H., Sinkovits, R. S., Potter, M., et al. (2012) · Journal of Virology · doi:10.1128/jvi.07232-11 · Cited by 219 · see citing works

The 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

Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types

Kumar, S. R., Miles, T. F., Chen, X., Brown, D. A., Dobreva, T., Huang, Q., Ding, X., Luo, Y., Einarsson, P. H., Greenbaum, A., et al. (2020) · Nature Methods · doi:10.1038/s41592-020-0799-7 · Cited by 194 · see citing works

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain

Matsuzaki, Y., Konno, A., Mochizuki, R., Shinohara, Y., Nitta, K., Okada, Y., & Hirai, H. (2018) · Neuroscience Letters · doi:10.1016/j.neulet.2017.11.049 · Cited by 173 · see citing works

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

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., et al. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.10.007 · Cited by 148 · see citing works

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

Liguore, W. A., Domire, J. S., Button, D. A., Wang, Y., Dufour, B. D., Srinivasan, S., & McBride, J. L. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.07.017 · Cited by 148 · see citing works

Neuron-specific Gene Expression of Synapsin I

Schoch, S., Cibelli, G., & Thiel, G. (1996) · Journal of Biological Chemistry · doi:10.1074/jbc.271.6.3317 · Cited by 147 · see citing works

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Hinderer, C., Bell, P., Katz, N., Vite, C. H., Louboutin, J.-P., Bote, E., Yu, H., Zhu, Y., Casal, M. L., Bagel, J., et al. (2018) · Human Gene Therapy · doi:10.1089/hum.2017.026 · Cited by 141 · see citing works

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

Hinderer, C. J., Bell, P. L., Vite, C. H., Louboutin, J., Grant, R. L., Bote, E., Yu, H., Pukenas, B. A., Hurst, R. W., & Wilson, J. M. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2014.51 · Cited by 129 · see citing works

Viral Vectors and Delivery Strategies for CNS Gene Therapy

Gray, S. J., Woodard, K., & Samulski, R. J. (2010) · Therapeutic Delivery · doi:10.4155/tde.10.50 · Cited by 124 · see citing works

The protelomerase of temperate Escherichia coli phage N15 has cleaving-joining activity

Deneke, J., Ziegelin, G., Lurz, R., & Lanka, E. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.14.7721 · Cited by 103 · see citing works

In vivo selection of a computationally designed SCHEMA AAV library yields a novel variant for infection of adult neural stem cells in the SVZ

Ojala, D. S., Sun, S., Santiago-Ortiz, J. L., Shapiro, M. G., Romero, P. A., & Schaffer, D. V. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.006 · Cited by 97 · see citing works

Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries

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

Helper-free Production of Laboratory Grade AAV and Purification by Iodixanol Density Gradient Centrifugation

Crosson, S. M., Dib, P., Smith, J. K., & Zolotukhin, S. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.05.001 · Cited by 81 · see citing works

Successful Expansion but Not Complete Restriction of Tropism of Adeno-Associated Virus by In Vivo Biopanning of Random Virus Display Peptide Libraries

Michelfelder, S., Kohlschütter, J., Skorupa, A., Pfennings, S., Müller, O. J., Kleinschmidt, J. A., & Trepel, M. (2009) · PLoS ONE · doi:10.1371/journal.pone.0005122 · Cited by 75 · see citing works

AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo

Powell, S., Samulski, R. J., & McCown, T. J. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.03.007 · Cited by 70 · see citing works

A MicroRNA124 Target Sequence Restores Astrocyte Specificity of gfaABC1D-Driven Transgene Expression in AAV-Mediated Gene Transfer

Taschenberger, G., Tereshchenko, J., & Kügler, S. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.03.009 · Cited by 54 · see citing works

Adeno-Associated Virus RNAs Appear in a Temporal Order and Their Splicing Is Stimulated during Coinfection with Adenovirus

Mouw, M. B., & Pintel, D. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.21.9878-9888.2000 · Cited by 53 · see citing works

A Comprehensive RNA Sequencing Analysis of the Adeno-Associated Virus (AAV) Type 2 Transcriptome Reveals Novel AAV Transcripts, Splice Variants, and Derived Proteins

Stutika, C., Gogol‐Döring, A., Botschen, L., Mietzsch, M., Weger, S., Feldkamp, M., Chen, W., & Heilbronn, R. (2015) · Journal of Virology · doi:10.1128/jvi.02750-15 · Cited by 42 · see citing works

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries

Schmit, P. F., Pacouret, S., Zinn, E. M., Telford, E., Nicolaou, F., Broucque, F., Andrés‐Mateos, E., Xiao, R., Penaud‐Budloo, M., Bouzelha, M., et al. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.014 · Cited by 34 · 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

Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates

Ellsworth, J. L., Gingras, J., Smith, L. J., Rubin, H. L., Seabrook, T. A., Patel, K., Zapata, N., Olivieri, K. C., O’Callaghan, M., Chlipala, E. A., et al. (2019) · PLoS ONE · doi:10.1371/journal.pone.0225582 · Cited by 25 · see citing works

Optimization of design and production strategies for novel adeno-associated viral display peptide libraries

Körbelin, J., Hunger, A., Alawi, M., Sieber, T., Binder, M., & Trepel, M. (2017) · Gene Therapy · doi:10.1038/gt.2017.51 · Cited by 19 · see citing works

How to Successfully Screen Random Adeno-Associated Virus Display Peptide Libraries In Vivo

Körbelin, J., & Trepel, M. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.177 · Cited by 11 · see citing works

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. F., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 895 · see citing works

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

Tabebordbar, M., Lagerborg, K. A., Stanton, A. C., King, E. M., Ye, S. H., Tellez, L., Krunnfusz, A., Tavakoli, S., Widrick, J. J., Messemer, K. A., et al. (2021) · Cell · doi:10.1016/j.cell.2021.08.028 · Cited by 517 · see citing works

AAV vectors: The Rubik’s cube of human gene therapy

Pupo, A., Fernández, A., Low, S. H., François, A., Suárez-Amarán, L., & Samulski, R. J. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.09.015 · Cited by 361 · 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

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

Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain

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

Direct neuronal reprogramming: Fast forward from new concepts toward therapeutic approaches

Bocchi, R., Masserdotti, G., & Götz, M. (2021) · Neuron · doi:10.1016/j.neuron.2021.11.023 · Cited by 128 · 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

Variants of the adeno-associated virus serotype 9 with enhanced penetration of the blood–brain barrier in rodents and primates

Yao, 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

A high-efficiency AAV for endothelial cell transduction throughout the central nervous system

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

Gene therapy clinical trials worldwide to 2023—an update

Ginn, S., Mandwie, M., Alexander, I. E., Edelstein, M. L., & Abedi, M. R. (2024) · Journal of Gene Medicine · doi:10.1002/jgm.3721 · Cited by 80 · see citing works

AAV vector development, back to the future

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 92 · see citing works

Fantastic AAV Gene Therapy Vectors and How to Find Them—Random Diversification, Rational Design and Machine Learning

Becker, J., Fakhiri, J., & Grimm, D. (2022) · Pathogens · doi:10.3390/pathogens11070756 · Cited by 80 · see citing works

Targeting AAV vectors to the central nervous system by engineering capsid–receptor interactions that enable crossing of the blood–brain barrier

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

Aggregate-selective removal of pathological tau by clustering-activated degraders

Benn, J., Cheng, S., Keeling, S., Smith, A. E., Vaysburd, M. J., Böken, D., Miller, L. V. C., Katsinelos, T., Franco, C. D. M. F., Dupré, E., et al. (2024) · Science · doi:10.1126/science.adp5186 · Cited by 76 · 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

Systematic multi-trait AAV capsid engineering for efficient gene delivery

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

CD33 isoforms in microglia and Alzheimer's disease: Friend and foe

Eskandari‐Sedighi, G., Jung, J., & Macauley, M. S. (2022) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2022.101111 · Cited by 68 · see citing works

A Multifaceted Approach to Optimizing AAV Delivery to the Brain for the Treatment of Neurodegenerative Diseases

Fischell, J. M., & Fishman, P. S. (2021) · Frontiers in Neuroscience · doi:10.3389/fnins.2021.747726 · Cited by 66 · see citing works

Primate-conserved carbonic anhydrase IV and murine-restricted LY6C1 enable blood-brain barrier crossing by engineered viral vectors

(2023) · doi:10.1126/sciadv.adg6618 · Cited by 67 · see citing works

New Drug Delivery Systems Developed for Brain Targeting

Rawal, S. U., Patel, B. M., & Patel, M. M. (2022) · Drugs · doi:10.1007/s40265-022-01717-z · Cited by 65 · see citing works

Adeno-Associated Virus Vector for Central Nervous System Gene Therapy

Zhu, D., Schieferecke, A. J., Lopez, P. A., & Schaffer, D. V. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2021.03.010 · Cited by 56 · see citing works

Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS

Stanton, A. C., Lagerborg, K. A., Tellez, L., Krunnfusz, A., King, E. M., Ye, S. H., Solomon, I. H., Tabebordbar, M., & Sabeti, P. C. (2022) · Med · doi:10.1016/j.medj.2022.11.002 · Cited by 61 · see citing works

AAV Engineering for Improving Tropism to the Central Nervous System

Ghauri, M. S., & Ou, L. (2023) · Biology · doi:10.3390/biology12020186 · Cited by 61 · see citing works

Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights

López-Gordo, E., Chamberlain, K., Riyad, J. M., Kohlbrenner, E., & Weber, T. (2024) · Viruses · doi:10.3390/v16030442 · Cited by 61 · see citing works

Co-opting templated aggregation to degrade pathogenic tau assemblies and improve motor function

Miller, L. V. C., Papa, G., Vaysburd, M., Cheng, S., Sweeney, P. W., Smith, A. N., Franco, C. D. M. F., Katsinelos, T., Huang, M., Sanford, S., et al. (2024) · Cell · doi:10.1016/j.cell.2024.08.024 · Cited by 58 · see citing works

A comprehensive atlas of AAV tropism in the mouse

Walkey, C. J., Snow, K. J., Bulcha, J., Cox, A. R., Martinez, A. E., Ljungberg, M. C., Lanza, D. G., Giorgi, M. D., Chuecos, M. A., Alves‐Bezerra, M., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.01.041 · Cited by 60 · see citing works

Spatial transcriptomics for profiling the tropism of viral vectors in tissues

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

Viral Tools for Neural Circuit Tracing

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

Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside

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

Kinetics and durability of transgene expression after intrastriatal injection of AAV9 vectors

Hollidge, B. S., Carroll, H. B., Qian, R., Fuller, M. L., Giles, A. R., Mercer, A. C., Danos, O., Liu, Y., Bruder, J. T., & Smith, J. B. (2022) · Frontiers in Neurology · doi:10.3389/fneur.2022.1051559 · Cited by 45 · see citing works

Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier

Moyer, T. C., Hoffman, B. A., Chen, W., Shah, I. S., Ren, X., Knox, T. M., Liu, J., Wang, W., Li, J., Khalid, H., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.04.046 · Cited by 58 · see citing works

New approaches to moderate CRISPR-Cas9 activity: Addressing issues of cellular uptake and endosomal escape

van Hees, M., Slott, S., Hansen, A. H., Kim, H. S., Ji, H. P., & Astakhova, K. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.06.003 · Cited by 46 · see citing works

Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

Pierce, G. F., Fong, S., Long, B. R., & Kaczmarek, R. (2023) · Journal of Thrombosis and Haemostasis · doi:10.1016/j.jtha.2023.12.005 · Cited by 45 · see citing works

Advances in AAV technology for delivering genetically encoded cargo to the nonhuman primate nervous system

Campos, L., Arokiaraj, C. M., Chuapoco, M. R., Chen, X., Goeden, N., Gradinaru, V., & Fox, A. S. (2023) · Current Research in Neurobiology · doi:10.1016/j.crneur.2023.100086 · Cited by 41 · see citing works

Receptor–mediated transcytosis of macromolecules across the blood–brain barrier

Baghirov, H. (2023) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2023.2255138 · Cited by 43 · see citing works

Current advances in gene therapy of mitochondrial diseases

Soldatov, V. O., Kubekina, M. V., Skorkina, M. Y., Белых, А. Е., Egorova, T. V., Корокин, М. В., Pokrovskiy, M. V., Deykin, A. V., & Angelova, P. R. (2022) · Journal of Translational Medicine · doi:10.1186/s12967-022-03685-0 · Cited by 41 · see citing works

Brain-targeting drug delivery systems: The state of the art in treatment of glioblastoma

Sun, B., Li, R., Ji, N., Liu, H., Wang, H., Chen, C., Bai, L., Su, J., & Chen, J. (2025) · Materials Today Bio · doi:10.1016/j.mtbio.2025.101443 · Cited by 39 · see citing works

From Lab Bench to Hope: Emerging Gene Therapies in Clinical Trials for Alzheimer’s Disease

Ortega, A., Chernicki, B. P., Ou, G., & Parmar, M. S. (2024) · Molecular Neurobiology · doi:10.1007/s12035-024-04285-3 · Cited by 41 · see citing works

Routes of administration for adeno-associated viruses carrying gene therapies for brain diseases

Zhou, K., Han, J., Wang, Y., Zhang, Y., & Zhu, C. (2022) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2022.988914 · Cited by 38 · see citing works

Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery

Gonzalez, T. J., Mitchell-Dick, A., Blondel, L. O., Fanous, M. M., Hull, J. A., Oh, D. K., Møller-Tank, S., Rivera, R. M. C., Piedrahita, J. A., & Asokan, A. (2023) · Nature Protocols · doi:10.1038/s41596-023-00875-y · Cited by 39 · see citing works

Liver directed adeno‐associated viral vectors to treat metabolic disease

Chuecos, M. A., & Lagor, W. R. (2023) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.12637 · Cited by 33 · see citing works

Adeno-Associated Virus Engineering and Load Strategy for Tropism Modification, Immune Evasion and Enhanced Transgene Expression

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

Artificial Intelligence‐Based Approaches for AAV Vector Engineering

Tan, F., Dong, Y., Qi, J., Yu, W., & Chai, R. (2025) · Advanced Science · doi:10.1002/advs.202411062 · Cited by 35 · see citing works

Spatial genomics of AAV vectors reveals mechanism of transcriptional crosstalk that enables targeted delivery of large genetic cargo

Coughlin, G. M., Borsos, M., Barcelona, B. H., Appling, N., Mayfield, A. M. H., Mackey, E. D. W., Eser, R. A., Jackson, C. R., Chen, X., Kumar, S. R., et al. (2025) · Nature Biotechnology · doi:10.1038/s41587-025-02565-4 · Cited by 28 · see citing works

Methods and applications of in vivo CRISPR screening

Santinha, A. J., Strano, A., & Platt, R. J. (2025) · Nature Reviews Genetics · doi:10.1038/s41576-025-00873-8 · Cited by 29 · see citing works

AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression

Westhaus, A., Cabanes‐Creus, M., Jonker, T., Sallard, E., Navarro, R. G., Zhu, E., Torres, G. B., Lee, S. D., Wilmott, P., Gonzalez-Cordero, A., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2021.278 · Cited by 28 · see citing works

Enhancer AAVs for targeting spinal motor neurons and descending motor pathways in rodents and macaque

Kussick, E., Johansen, N., Taskin, N., Chowdhury, A., Quinlan, M. A., Fraser, A., Clark, A. G., Wynalda, B., Martinez, R. A., Groce, E. L., et al. (2025) · Cell Reports · doi:10.1016/j.celrep.2025.115730 · Cited by 27 · see citing works

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Davidson, B. L., Gao, G., Berry‐Kravis, E., Bradbury, A. M., Bönnemann, C. G., Buxbaum, J. D., Corcoran, G. R., Gray, S. J., Gray‐Edwards, H. L., Kleiman, R. J., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.05.014 · Cited by 26 · see citing works

Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles

Buñuales, M., Garduno, A., Chillón, M., Bosch, A., González-Aparicio, M., Espelosin, M., García-Gómara, M., Rico, A. J., García‐Osta, A., Cuadrado‐Tejedor, M., et al. (2024) · Gene Therapy · doi:10.1038/s41434-024-00466-w · Cited by 26 · see citing works

Directed evolution of adeno-associated virus 5 capsid enables specific liver tropism

Wang, Y., Yang, C., Hu, H., Chen, C., Yan, M., Ling, F., Wang, K. C., Wang, X., Deng, Z., Zhou, X., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.03.017 · Cited by 26 · see citing works

An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature

Ramirez, S. H., Hale, J. F., McCarthy, S., Cardenas, C. L. L., Dona, K. N. U. G., Hanlon, K. S., Hudry, E., de la Cruz, D., Ng, C., Das, S., et al. (2023) · Human Gene Therapy · doi:10.1089/hum.2022.211 · Cited by 25 · see citing works

Receptor Switching in Newly Evolved Adeno-associated Viruses

Havlik, L. P., Das, A., Mietzsch, M., Oh, D. K., Ark, J., McKENNA, R. V., Agbandje‐McKenna, M., & Asokan, A. (2021) · Journal of Virology · doi:10.1128/jvi.00587-21 · Cited by 24 · see citing works

AAV-vector based gene therapy for mitochondrial disease: progress and future perspectives

Hanaford, A. R., Cho, Y., & Nakai, H. (2022) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-022-02324-7 · Cited by 23 · see citing works

Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier

Critchley, B. J., Gaspar, H. B., & Benedetti, S. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.11.015 · Cited by 23 · see citing works

Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid

Goertsen, D., Goeden, N., Flytzanis, N. C., & Gradinaru, V. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.07.010 · Cited by 23 · see citing works

Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors

Liu, J., Koay, T. W., Maiakovska, O., Zayas, M., & Grimm, D. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.057 · Cited by 22 · see citing works

Molecular Engineering of Virus Tropism

He, B., Wilson, B., Chen, S., Sharma, K., Scappini, E., Cook, M., Petrovich, R. M., & Martin, N. P. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms252011094 · Cited by 21 · see citing works

Engineering novel adeno-associated viruses (AAVs) for improved delivery in the nervous system

Carneiro, A. D., & Schaffer, D. V. (2024) · Current Opinion in Chemical Biology · doi:10.1016/j.cbpa.2024.102532 · Cited by 21 · see citing works

Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling

Ding, X., Chen, X., Sullivan, E. E., Shay, T. F., & Gradinaru, V. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.04.003 · Cited by 20 · see citing works

Enhancer-AAVs allow genetic access to oligodendrocytes and diverse populations of astrocytes across speciesPreprint

Mich, J. K., Sunil, S., Johansen, N., Martinez, R. A., Liu, J., Gore, B. B., Mahoney, J. T., Leytze, M., Ben‐Simon, Y., Bertagnolli, D., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.09.20.558718 · Cited by 21 · see citing works

Next-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

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

From lab bench to hope: a review of gene therapies in clinical trials for Parkinson’s disease and challenges

Grote, J., Patel, N. J., Bates, C. M., & Parmar, M. S. (2024) · Neurological Sciences · doi:10.1007/s10072-024-07599-1 · Cited by 19 · see citing works

Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors

Macdonald, J., Marx, J., & Büning, H. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.169 · Cited by 16 · see citing works

A comprehensive study of AAV tropism across C57BL/6 mice, BALB/c mice, and crab-eating macaques

Fang, K., Yang, X., Liu, Y., Xia, J., Wu, R., Yang, F., Feng, C., Liu, X., Shi, L., Geng, G., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101434 · Cited by 16 · see citing works

Long-term effects of s-KL treatment in wild-type mice: Enhancing longevity, physical well-being, and neurological resilience

Roig-Soriano, J., Edo, Á., Verdés, S., Martín-Alonso, C., Sánchez‐de‐Diego, C., Rodríguez-Estévez, L., Serrano, A. L., Abraham, C. R., Bosch, A., Ventura, F., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.02.030 · Cited by 16 · see citing works

Progress in AAV-Mediated In Vivo Gene Therapy and Its Applications in Central Nervous System Diseases

Wang, S., & Xiao, L. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26052213 · Cited by 15 · see citing works

Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice

Chang, H., Du, A., Jiang, J., Ren, L., Liu, N., Zhou, X., Liang, J., Gao, G., & Wang, D. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101129 · Cited by 14 · see citing works

Structural basis of the recognition of adeno-associated virus by the neurological system-related receptor carbonic anhydrase IV

Zhang, R., Liu, Y., Yu, F., Xu, G., Li, L., Li, B., & Lou, Z. (2024) · PLoS Pathogens · doi:10.1371/journal.ppat.1011953 · Cited by 13 · see citing works

Adeno-Associated Virus Delivery Limitations for Neurological Indications

Benatti, H. R., & Gray‐Edwards, H. L. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.29196.hrb · Cited by 14 · see citing works

In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord

Hanlon, 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

Emerging Gene Therapies for Alzheimer’s and Parkinson’s Diseases: An Overview of Clinical Trials and Promising Candidates

Roberts, W. S., Price, S., Wu, M., & Parmar, M. S. (2024) · Cureus · doi:10.7759/cureus.67037 · Cited by 16 · see citing works

An engineered adeno-associated virus mediates efficient blood-brain barrier penetration with enhanced neurotropism and reduced hepatotropism

Luo, 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

Highly conserved brain vascular receptor ALPL mediates transport of engineered viral vectors across the blood-brain barrierPreprint

Moyer, T. C., Hoffman, B. A., Chen, W., Shah, I. S., Ren, X., Knox, T. M., Liu, J., Wang, W., Li, J., Khalid, H., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.03.12.584703 · Cited by 13 · see citing works

Gene and Cellular Therapies for Leukodystrophies

Aerts‐Kaya, F., & van Til, N. P. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15112522 · Cited by 13 · see citing works

AAV vectors for specific and efficient gene expression in microglia

Aoki, R., Konno, A., Hosoi, N., Kawabata, H., & Hirai, H. (2025) · Cell Reports Methods · doi:10.1016/j.crmeth.2025.101116 · Cited by 12 · see citing works

AAV-mediated interneuron-specific gene replacement for Dravet syndromePreprint

Mich, J. K., Ryu, J., Wei, A. D., Gore, B. B., Guo, R., Bard, A. M., Martinez, R. A., Bishaw, Y. M., Luber, E. M., Oliveira, L. M., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.12.15.571820 · Cited by 12 · 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

Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library

Becker, J., Domenger, C., Choksi, P. K., Krämer, C., Baumgartl, C., Maiakovska, O., Kim, J.-J., Weinmann, J., Huber, G., Schmidt, F., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.04.027 · Cited by 11 · see citing works

Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid

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

Gene therapy for Parkinson’s disease: a new frontier in neurodegenerative diseases

Asuku, A. O., Ogungbangbe, G. O., & Fajemidagba, G. A. (2025) · Exploration of Neuroprotective Therapy · doi:10.37349/ent.2025.1004119 · Cited by 10 · see citing works

A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice

Sangster, M., Bishop, M., Yao, Y., Feitor, J. F., Shahriar, S., Miller, M. E., Chekuri, A. K., Budnik, B., Bei, F., & Grishchuk, Y. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101269 · Cited by 10 · see citing works

Identification of new AAV vectors with enhanced blood–brain barrier penetration efficiency via organ-on-a-chip

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

Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation

Westhaus, A., Barba-Sarasua, E., Chen, Y., Hsu, K., Scott, S., Knight, M., Haase, F., Mora, S. M., Houghton, B. C., Roca-Pinilla, R., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.12.012 · Cited by 10 · see citing works

Peripherally delivered Adeno-associated viral vectors for spinal cord injury repair

Sydney-Smith, J. D., Spejo, A. B., Warren, P. M., & Moon, L. D. F. (2021) · Experimental Neurology · doi:10.1016/j.expneurol.2021.113945 · Cited by 10 · see citing works

Novel AAV variants with improved tropism for human Schwann cells

Drouyer, M., Chu, T., Labit, E., Haase, F., Navarro, R. G., Nazareth, D., Rosin, N. L., Merjane, J., Scott, S., Cabanes‐Creus, M., et al. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101234 · Cited by 10 · see citing works

Tissue 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

AAVs targeting human carbonic anhydrase IV enhance gene delivery to the brain

Lin, C., Chen, X., Hoang, J. D., Ristic, F., Fan, Y., Jang, S., Chung, J. H. A., Sullivan, E. E., Gawda, T., Kavvathas, B., et al. (2025) · Cell Reports · doi:10.1016/j.celrep.2025.116419 · Cited by 8 · see citing works

Adeno-associated viral vectors for modeling Parkinson’s disease in non-human primates

Chocarro, J., & Lanciego, J. L. (2025) · Neural Regeneration Research · doi:10.4103/nrr.nrr-d-24-00896 · Cited by 8 · see citing works

Beyond the blood–brain barrier: the fate of transcytosed therapeutics

Baghirov, H. (2025) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2025.08.006 · 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

The amazing AAV capsids: Into the structure-verse

Hsi, J., Mietzsch, M., & McKENNA, R. V. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101629 · Cited by 8 · see citing works

Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency

Du, A., Yang, K., Zhou, X.-T., Ren, L., Liu, N., Zhou, C., Liang, J., Yan, N., Gao, G.-P., & Wang, D. (2024) · JCI Insight · doi:10.1172/jci.insight.183189 · Cited by 4 · see citing works

Characterization of a Bioengineered AAV3B Capsid Variant with Enhanced Hepatocyte Tropism and Immune Evasion

Rana, J., Marsic, D., Zou, C., Muñoz-Melero, M., Li, X., Kondratov, O., Li, N., de Jong, Y. P., Zolotukhin, S., & Biswas, M. (2023) · Human Gene Therapy · doi:10.1089/hum.2022.176 · Cited by 7 · see citing works

Directed evolution of novel AAV variants using the MCMS library for enhanced CNS tropism and reduced liver targeting in mice

Song, Q., Wu, J., Zhang, Q., Wu, S. V., Luo, X., Xu, M., Xu, Y., Wang, G., Yu, H., Chen, H., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101522 · Cited by 7 · see citing works

Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates

Okai, T., Sato, S., Yasuno, H., Nakayama, M., Yamamoto, S., Sjöqvist, S., Otake, K., Nakashima, M., Deshpande, M., Galbreath, E. J., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101593 · Cited by 7 · see citing works

A targeted vector for brain endothelial cell gene delivery and cerebrovascular malformation modelling

Li, J.-L., Bi, Z., Chen, X.-J., Ming, T., Qiu, B., Li, F., Feng, Z., Ai, D., Zhang, T., Wang, J., et al. (2025) · Nature Biomedical Engineering · doi:10.1038/s41551-025-01538-x · Cited by 6 · see citing works

Advances in gene therapy for Lafora disease: Intravenous recombinant adeno‐associated virus‐mediated delivery of EPM2A and EPM2B genes

Zafra‐Puerta, L., Iglesias‐Cabeza, N., Sciaccaluga, M., Bellingacci, L., Canonichesi, J., Sánchez‐Martín, G., Costa, C., Sánchez, M. P., & Serratosa, J. M. (2025) · Clinical and Translational Medicine · doi:10.1002/ctm2.70514 · Cited by 6 · see citing works

AAV Engineering for Improving Tropism to the Central Nervous System

Ghauri, M. S., & Ou, L. (2023) · Biology · doi:10.3390/biology12020186 · Cited by 61 · see citing works

Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice

Drouyer, M., Merjane, J., Nazareth, D., Knight, M., Scott, S., Liao, S. H. Y., Ginn, S. L., Zhu, E., Alexander, I. E., & Lisowski, L. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.01.024 · Cited by 17 · see citing works

Targeting AAV vectors to the central nervous system by engineering capsid–receptor interactions that enable crossing of the blood–brain barrier

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

Directed evolution of novel AAV variants using the MCMS library for enhanced CNS tropism and reduced liver targeting in mice

Song, Q., Wu, J., Zhang, Q., Wu, S. V., Luo, X., Xu, M., Xu, Y., Wang, G., Yu, H., Chen, H., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101522 · Cited by 7 · see citing works

Evolution and Investigation of Engineered AAV Capsids Exhibiting Enhanced Transduction of the Central Nervous System with or without Murine Strain Specificity

Kumar, S. R., Miles, T. F., Chen, X., Brown, D. A., Dobreva, T., Huang, Q., Ding, X., Luo, Y., Einarsson, P. H., Greenbaum, A., et al. (2020)