Capsid modifications for targeting and improving the efficacy of AAV vectors

Büning, Hildegard; Srivastava, Arun

doi:10.1016/j.omtm.2019.01.008

Cited by 260Virus-based gene therapy researchRNA Interference and Gene DeliveryParvovirus B19 Infection Studies

Capsid modifications for targeting and improving the efficacy of AAV vectors

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Büning, Hildegard, Srivastava, Arun·2019·Molecular Therapy - Methods & Clinical Development

DOI 10.1016/j.omtm.2019.01.008PMC PMC6378346

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Abstract

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical trials and their efficacy in at least eight human diseases are now firmly documented. Despite these remarkable achievements, it has also become abundantly clear that the full potential of first generation AAV vectors composed of naturally occurring capsids is not likely to be realized, since the wild-type AAV did not evolve for the purpose of therapeutic gene delivery. In this article, we provide a brief historical account of the progress that has been made in the development of capsid-modified , next-generation AAV vectors to ensure both the safety and efficacy of these vectors in targeting a wide variety of human diseases.

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Engineered vector properties can cloak the viral vector from or modulate the innate and adaptive immune responses.
“Secondly, engineered vector properties can 'cloak ' the viral vector from (or modulate) the innate and adaptive immune responses (Büning and Srivastava, 2019; Prasad et al., 2022).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Büning and Srivastava, 2019
The Conclusions (page not provided) say capsid modifications can alter the host immune response and give examples of reduced CD8+ T-cell responses and partial evasion of pre-existing antibodies. These support modulation or evasion of adaptive immunity, but the passages do not establish an effect on innate immune responses. (LLM, conf=0.85)
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Successful target cell transduction of capsid-engineered rAAV vectors requires clathrin-dependent endocytosis
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Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV- GAA ) Gene Vector in Patients with Pompe Disease
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Insertional Mutagenesis at Positions 520 and 584 of Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Generation of AAV2 Vectors with Eliminated Heparin- Binding Ability and Introduced Novel Tropism
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Modular adeno-associated virus (rAAV) vectors used for cellular virus-directed enzyme prodrug therapy
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AAV's Golden Jubilee
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Proof of concept study with an HER-2 mimotope anticancer vaccine deduced from a novel AAV-mimotope library platform
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Application of a haematopoetic progenitor cell-targeted adeno-associated viral (AAV) vector established by selection of an AAV random peptide library on a leukaemia cell line
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Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In Vivo
Geoghegan, J. C., Keiser, N. W., Okulist, A., Martins, I., Wilson, M. S., & Davidson, B. L. (2014) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2014.50 · Cited by 14 · see citing works
A Novel Directed Evolution Method to Enhance Cell-Type Specificity of Adeno-Associated Virus Vectors
Marsch, S. C. U., Huber, A., Hallek, M. J., Büning, H., & Perabò, L. (2010) · Combinatorial Chemistry & High Throughput Screening · doi:10.2174/138620710792927385 · Cited by 14 · see citing works
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Impact of capsid modifications by selected peptide ligands on recombinant adeno-associated virus serotype 2-mediated gene transduction
Naumer, M., Popa-Wagner, R., & Kleinschmidt, J. A. (2012) · Journal of General Virology · doi:10.1099/vir.0.044735-0 · Cited by 13 · see citing works
Immobilization of FLAG-Tagged Recombinant Adeno-Associated Virus 2 onto Tissue Engineering Scaffolds for the Improvement of Transgene Delivery in Cell Transplants
Li, H., Zhang, F., Shi, W.-J., Bai, X.-J., Jia, S.-Q., Zhang, C., & Ding, W. (2015) · PLoS ONE · doi:10.1371/journal.pone.0129013 · Cited by 13 · see citing works
A chemical switch for controlling viral infectivity
Hörner, M., Kaufmann, B., Cotugno, G., Wiedtke, E., Büning, H., Grimm, D., & Weber, W. (2014) · Chemical Communications · doi:10.1039/c4cc03292f · Cited by 10 · see citing works
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Advances and challenges in the use of recombinant AAV vectors for human gene therapy
Srivastava, A. (2016) · Cell and Gene Therapy Insights · doi:10.18609/cgti.2016.061 · Cited by 8 · see citing works
Self-citation
Adeno‐associated Viral Vectors in Gene Therapy
Ling, C., Zhong, L., & Srivastava, A. (2018) · Wiley eBooks · doi:10.1002/9780470015902.a0005738.pub3 · Cited by 4 · see citing works
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Pulmonary delivery of siRNA against acute lung injury/acute respiratory distress syndrome
Zoulikha, M., Xiao, Q., Boafo, G. F., Sallam, M. A., Chen, Z., & He, W. (2021) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2021.08.009 · Cited by 285 · see citing works
Use of Cell and Genome Modification Technologies to Generate Improved “Off-the-Shelf” CAR T and CAR NK Cells
Morgan, M. A., Büning, H., Sauer, M. G., & Schambach, A. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.01965 · Cited by 151 · see citing works
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Novel vectors and approaches for gene therapy in liver diseases
Maestro, S., Weber, N., Zabaleta, N., Aldabe, R., & González-Aseguinolaza, G. (2021) · JHEP Reports · doi:10.1016/j.jhepr.2021.100300 · Cited by 117 · see citing works
SARM1 acts downstream of neuroinflammatory and necroptotic signaling to induce axon degeneration
Ko, K. W., Milbrandt, J., & DiAntonio, A. (2020) · The Journal of Cell Biology · doi:10.1083/jcb.201912047 · Cited by 133 · see citing works
Journey to the Center of the Cell: Tracing the Path of AAV Transduction
Dhungel, B. P., Bailey, C. G., & Rasko, J. E. J. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2020.09.010 · Cited by 122 · see citing works
Precision medicine: In vivo CAR therapy as a showcase for receptor-targeted vector platforms
Michels, A., Ho, N., & Buchholz, C. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.05.018 · Cited by 110 · see citing works
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
Riyad, J. M., & Weber, T. (2021) · Gene Therapy · doi:10.1038/s41434-021-00243-z · Cited by 116 · 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 new age of precision gene therapy
Schambach, A., Buchholz, C. J., Torres-Ruiz, R., Cichutek, K., Morgan, M. A., Trapani, I., & Büning, H. (2023) · The Lancet · doi:10.1016/s0140-6736(23)01952-9 · Cited by 126 · see citing works
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Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper
Yang, T., Braun, M., Lembke, W., McBlane, F., Kamerud, J., DeWall, S. L., Tarcsa, E., Fang, X., Hofer, L., Kavita, U., et al. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.07.018 · Cited by 118 · see citing works
High-Throughput In Vitro , Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction
Westhaus, 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 · see citing works
Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors
Andari, J. E., & Grimm, D. (2020) · Biotechnology Journal · doi:10.1002/biot.202000025 · Cited by 97 · 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
Evolving a Peptide: Library Platforms and Diversification Strategies
Bozovičar, K., & Bratkovič, T. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms21010215 · Cited by 81 · see citing works
Critical challenges and advances in recombinant adeno‐associated virus (rAAV) biomanufacturing
Fu, Q., Polanco, A., Lee, Y. S., & Yoon, S. (2023) · Biotechnology and Bioengineering · doi:10.1002/bit.28412 · Cited by 82 · 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
Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors
Achberger, K., Cipriano, M., Duechs, M. J., Schön, C., Michelfelder, S., Stierstorfer, B., Lamla, T., Kauschke, S. G., Chuchuy, J., Roosz, J., et al. (2021) · Stem Cell Reports · doi:10.1016/j.stemcr.2021.08.008 · Cited by 73 · see citing works
Enzymatic Bioconjugation: A Perspective from the Pharmaceutical Industry
Debon, A., Siirola, E., & Šnajdrová, R. (2023) · JACS Au · doi:10.1021/jacsau.2c00617 · Cited by 74 · see citing works
Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening
Börner, K., Kienle, E., Huang, L., Weinmann, J., Sacher, A., Bayer, P., Stüllein, C., Fakhiri, J., Zimmermann, L., Westhaus, A., et al. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.02.009 · Cited by 72 · see citing works
Towards Clinical Implementation of Adeno-Associated Virus (AAV) Vectors for Cancer Gene Therapy: Current Status and Future Perspectives
Hacker, U. T., Bentler, M., Kaniowska, D., Morgan, M. A., & Büning, H. (2020) · Cancers · doi:10.3390/cancers12071889 · Cited by 71 · see citing works
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In Vivo Delivery of Nucleic Acid-Encoded Monoclonal Antibodies
Patel, A. R., Bah, M. A., & Weiner, D. B. (2020) · BioDrugs · doi:10.1007/s40259-020-00412-3 · Cited by 70 · see citing works
Bacteriophage Capsid Modification by Genetic and Chemical Methods
Carmody, C. M., Goddard, J. M., & Nugen, S. R. (2021) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.1c00018 · 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
Evolving AAV-delivered therapeutics towards ultimate cures
He, X., Urip, B. A., Zhang, Z., Ngan, C. C., & Feng, B. (2021) · Journal of Molecular Medicine · doi:10.1007/s00109-020-02034-2 · Cited by 67 · see citing works
CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
Rahman, M. U., Bilal, M., Shah, J. A., Kaushik, A. K., Teissèdre, P., & Kujawska, M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14061252 · Cited by 65 · see citing works
AAV capsid design: A Goldilocks challenge
Zolotukhin, S., & Vandenberghe, L. H. (2022) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2022.01.003 · Cited by 64 · see citing works
Engineered mitochondria in diseases: mechanisms, strategies, and applications
Li, M., Wu, L., Si, H., Wu, Y., Liu, Y., Zeng, Y., & Shen, B. (2025) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-02081-y · Cited by 64 · 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
Materials promoting viral gene delivery
Kaygisiz, K., & Synatschke, C. V. (2020) · Biomaterials Science · doi:10.1039/d0bm01367f · Cited by 56 · see citing works
Muscle-Specific Promoters for Gene Therapy
Skopenkova, V. V., Egorova, T. V., & Bardina, M. V. (2021) · Acta Naturae · doi:10.32607/actanaturae.11063 · Cited by 57 · see citing works
Development of New Strategies Using Extracellular Vesicles Loaded with Exogenous Nucleic Acid
Orefice, N. S. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12080705 · Cited by 56 · see citing works
Click-electrochemistry for the rapid labeling of virus, bacteria and cell surfaces
Depienne, S., Bouzelha, M., Courtois, E., Pavageau, K., Lalys, P.-A., Marchand, M., Álvarez‐Dorta, D., Nédellec, S., Marín-Fernández, L., Grandjean, C., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-40534-0 · Cited by 51 · see citing works
Vector engineering, strategies and targets in cancer gene therapy
Singh, V., Khan, N., & Jayandharan, G. R. (2021) · Cancer Gene Therapy · doi:10.1038/s41417-021-00331-7 · Cited by 51 · see citing works
Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates
Samelson-Jones, B. J., Finn, J. D., Favaro, P., Wright, J. F., & Arruda, V. R. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.05.001 · Cited by 50 · see citing works
Ocular drug delivery: advancements and innovations
Tian, B., Bilsbury, E., Doherty, S., Teebagy, S., Wood, E., Su, W., Gao, G., & Lin, H. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091931 · Cited by 50 · see citing works
Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors
Bates, R., Huang, W., & Cao, L. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.09.009 · Cited by 49 · see citing works
Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy
Wagner, H. J., Weber, W., & Fussenegger, M. (2021) · Advanced Science · doi:10.1002/advs.202004018 · Cited by 48 · see citing works
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes
Rode, L., Bär, C., Groß, S., Rossi, A., Meumann, N., Viereck, J., Abbas, N., Xiao, K., Riedel, I., Gietz, A., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.07.003 · Cited by 48 · see citing works
Self-citation
Structural and cellular biology of adeno-associated virus attachment and entry
Zengel, J. R., & Carette, J. E. (2020) · Advances in virus research · doi:10.1016/bs.aivir.2020.01.002 · Cited by 44 · see citing works
CRISPR-Cas System: The Current and Emerging Translational Landscape
Bhokisham, N., Laudermilch, E., Traeger, L. L., Bonilla, T. D., Ruíz-Estévez, M., & Becker, J. R. (2023) · Cells · doi:10.3390/cells12081103 · Cited by 43 · see citing works
Process Improvement of Adeno-Associated Virus Production
Guan, J.-S., Chen, K., Si, Y., Kim, T. H., Zhou, Z. “., Kim, S., Zhou, L., & Liu, X. (2022) · Frontiers in Chemical Engineering · doi:10.3389/fceng.2022.830421 · Cited by 42 · see citing works
Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies
Stone, D., Aubert, M., & Jerome, K. R. (2023) · Gene Therapy · doi:10.1038/s41434-023-00405-1 · Cited by 41 · see citing works
Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates
Ballon, D., Rosenberg, J. B., Fung, E. K., Nikolopoulou, A., Kothari, P. J., De, B. P., He, B., Chen, A., Heier, L., Sondhi, D., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.116 · Cited by 37 · see citing works
Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea
Frederick, A., Sullivan, J., Liu, L., Adamowicz, M., Lukason, M., Raymer, J., Luo, Z., Jin, X., Rao, K. N., & O'Riordan, C. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.070 · Cited by 35 · see citing works
Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors
Verdera, H. C., Unzu, C., Valeri, E., Adriouch, S., González‐Aseguinolaza, G., Mingozzi, F., & Kajaste‐Rudnitski, A. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.119 · Cited by 40 · see citing works
Fast and high-throughput LC-MS characterization, and peptide mapping of engineered AAV capsids using LC-MS/MS
Lam, A. K., Zhang, J., Frabutt, D. A., Mulcrone, P. L., Li, L., Zeng, L., Herzog, R. W., & Xiao, W. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.09.008 · Cited by 38 · see citing works
An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species
Hong, A. V., Suel, L., Petat, E., Dubois, A., Brun, P., Guerchet, N., Veron, P., Poupiot, J., & Richard, I. (2024) · Nature Communications · doi:10.1038/s41467-024-52002-4 · Cited by 40 · see citing works
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects
Chwalenia, K., Feng, V.-Y., Hemmer, N., Friedrichsen, H. J., Vorobieva, I., Wood, M. J. A., & Roberts, T. C. (2025) · Gene Therapy · doi:10.1038/s41434-025-00561-6 · Cited by 37 · see citing works
Improved targeting of human CD4+ T cells by nanobody-modified AAV2 gene therapy vectors
Hamann, M. V., Beschorner, N., Vu, X., Hauber, I., Lange, U. C., Traenkle, B., Kaiser, P. D., Foth, D., Schneider, C., Büning, H., et al. (2021) · PLoS ONE · doi:10.1371/journal.pone.0261269 · Cited by 36 · see citing works
Self-citation
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8
Demircan, M. B., Zinser, L. J., Michels, A., Guaza-Lasheras, M., John, F., Gorol, J. M., Theuerkauf, S. A., Günther, D. M., Grimm, D., Greten, F. R., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.08.002 · Cited by 36 · see citing works
Genetic-Based Approaches to Inherited Metabolic Liver Diseases
Zabaleta, N., Hommel, M., Salas, D., & González‐Aseguinolaza, G. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.140 · Cited by 35 · see citing works
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone
Kremer, L. P. M., Cerrizuela, S., Dehler, S., Stiehl, T., Weinmann, J., Abendroth, H., Kleber, S., Laure, A., Andari, J. E., Anders, S., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.07.001 · Cited by 34 · see citing works
Recent advances in various adeno-associated viruses (AAVs) as gene therapy agents in hepatocellular carcinoma
Hadi, M., Allela, O. Q. B., Jabari, M., Jasoor, A. M., Naderloo, O., Yasamineh, S., Gholizadeh, O., & Kalantari, L. (2024) · Virology Journal · doi:10.1186/s12985-024-02286-1 · Cited by 35 · see citing works
Viral vectors for gene delivery to the inner ear
Maguire, C. A., & Corey, D. P. (2020) · Hearing Research · doi:10.1016/j.heares.2020.107927 · Cited by 32 · see citing works
Gene targeting as a therapeutic avenue in diseases mediated by the complement alternative pathway
Dreismann, A. K., Hallam, T. M., Tam, L. C. S., Nguyen, C. V., Hughes, J. P., Ellis, S., & Harris, C. L. (2022) · Immunological Reviews · doi:10.1111/imr.13149 · Cited by 33 · see citing works
Structure comparison of the chimeric AAV2.7m8 vector with parental AAV2
Bennett, A., Keravala, A., Makal, V., Kurian, J., Belbellaa, B., Aeran, R., Tseng, Y.-S., Sousa, D., Spear, J., Gasmi, M., et al. (2020) · Journal of Structural Biology · doi:10.1016/j.jsb.2019.107433 · Cited by 33 · see citing works
“, 2017)) towards enhancing transduction efficiency or immunological profile in vivo (Buning and Srivastava, 2019).”
Background
Modifying immune responses to adeno-associated virus vectors by capsid engineering
Bentler, M., Hardet, R., Ertelt, M., Rudolf, D., Kaniowska, D., Schneider, A., Vondran, F. W. R., Schoeder, C. T., Delphin, M., Lucifora, J., et al. (2023) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2023.08.015 · Cited by 34 · see citing works
Self-citation
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
Capsid Engineering Overcomes Barriers Toward Adeno-Associated Virus Vector-Mediated Transduction of Endothelial Cells
Zhang, L., Rossi, A., Lange, L., Meumann, N., Koitzsch, U., Christie, K. A., Nesbit, M. A., Moore, C. B. T., Hacker, U. T., Morgan, M. A., et al. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.027 · Cited by 33 · see citing works
Self-citation
Engineering Targeting Materials for Therapeutic Cancer Vaccines
Briquez, P. S., Hauert, S., de Titta, A., Gray, L. T., Alpar, A. T., Swartz, M. A., & Hubbell, J. A. (2020) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2020.00019 · Cited by 32 · 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
Promise and Progress of an HIV-1 Cure by Adeno-Associated Virus Vector Delivery of Anti-HIV-1 Biologics
Gardner, M. R. (2020) · Frontiers in Cellular and Infection Microbiology · doi:10.3389/fcimb.2020.00176 · Cited by 31 · see citing works
Trends and challenges of AAV-delivered gene editing therapeutics for CNS disorders: Implications for neurodegenerative disease
Kantor, B., O’Donovan, B., & Chiba‐Falek, O. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102635 · Cited by 36 · see citing works
Progress in Respiratory Gene Therapy
McLachlan, G., Alton, E., Boyd, A., Clarke, N. K., Davies, J., Gill, D., Griesenbach, U., Hickmott, J., Hyde, S., Miah, K., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.172 · Cited by 26 · see citing works
AAV vectors displaying bispecific DARPins enable dual-control targeted gene delivery
Theuerkauf, S. A., Herrera-Carrillo, E., John, F., Zinser, L. J., Molina, M. A., Riechert, V., Thalheimer, F. B., Börner, K., Grimm, D., Chlanda, P., et al. (2023) · Biomaterials · doi:10.1016/j.biomaterials.2023.122399 · Cited by 30 · see citing works
Computationally guided AAV engineering for enhanced gene delivery
Guo, J., Lin, L. F., Oraskovich, S. V., de Jesús, J. A. R., Listgarten, J., & Schaffer, D. V. (2024) · Trends in Biochemical Sciences · doi:10.1016/j.tibs.2024.03.002 · Cited by 30 · see citing works
Anti-ageing gene therapy: Not so far away?
Vaiserman, A. M., FALCO, E. D., Koliada, A. K., Maslova, O., & Balistreri, C. R. (2019) · Ageing Research Reviews · doi:10.1016/j.arr.2019.100977 · Cited by 30 · see citing works
DNA‐editing enzymes as potential treatments for heteroplasmic mtDNA diseases
Zekonyte, U., Bacman, S. R., & Moraes, C. T. (2020) · Journal of Internal Medicine · doi:10.1111/joim.13055 · Cited by 29 · see citing works
Strategies to improve safety profile of AAV vectors
Suoranta, T., Laham-Karam, N., & Ylä‐Herttuala, S. (2022) · Frontiers in Molecular Medicine · doi:10.3389/fmmed.2022.1054069 · Cited by 30 · 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
Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity
Biswas, M., Marsic, D., Li, N., Zou, C., González‐Aseguinolaza, G., Zolotukhin, I., Kumar, S. R. P., Rana, J., Butterfield, J. S. S., Kondratov, O., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.09.019 · Cited by 28 · see citing works
Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations
Drouyer, M., Merjane, J., Nedelkoska, T., Westhaus, A., Scott, S., Lee, S., Burke, P. G. R., McMullan, S., Lanciego, J. L., Vicente, A. F., et al. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102264 · Cited by 27 · see citing works
Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence
Olaghere, J., Williams, D. A., Farrar, J., Büning, H., Calhoun, C. L., Ho, T. W., Inamdar, M. S., Liu, D. R., Makani, J., Nyarko, K. A., et al. (2025) · Biomedicines · doi:10.3390/biomedicines13030758 · Cited by 27 · see citing works
Self-citation
Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy
Rodríguez-Márquez, E., Meumann, N., & Büning, H. (2020) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2021.1865303 · Cited by 27 · see citing works
Self-citation
The Structures and Functions of Parvovirus Capsids and Missing Pieces: the Viral DNA and Its Packaging, Asymmetrical Features, Nonprotein Components, and Receptor or Antibody Binding and Interactions
López-Astacio, R. A., Adu, O. F., Lee, H., Hafenstein, S. L., & Parrish, C. R. (2023) · Journal of Virology · doi:10.1128/jvi.00161-23 · Cited by 27 · see citing works
Clinical and Translational Landscape of Viral Gene Therapies
Yudaeva, A., Kostyusheva, A., Kachanov, A., Brezgin, S., Ponomareva, N., Parodi, A., Pokrovsky, V. S., Lukashev, A. N., Chulanov, V., & Kostyushev, D. S. (2024) · Cells · doi:10.3390/cells13221916 · Cited by 26 · see citing works
Comparative analysis of adeno-associated virus serotypes for gene transfer in organotypic heart slices
Liu, Z., Klose, K., Neuber, S., Jiang, M., Gossen, M., & Stamm, C. (2020) · Journal of Translational Medicine · doi:10.1186/s12967-020-02605-4 · Cited by 26 · see citing works
Directed evolution of adeno-associated virus 5 capsid enables specific liver tropism
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Authors: Büning, H., & Srivastava, A.
Affiliations: Medizinische Hochschule Hannover (DE)
University of Florida Health (US)
Corresponding author: Büning, Hildegard
Srivastava, Arun
ORCID: Büning, Hildegard
Srivastava, Arun
Venue: Molecular Therapy - Methods & Clinical Development
Publisher: Elsevier BV
Published: 2019-03
Volume: 12
Pages: 248-265
ISSN: 2329-0501 (ISSN-L)
DOI: 10.1016/j.omtm.2019.01.008
PMC: PMC6378346
OpenAlex: W2912684492
Cited by: 260
References: 147
Citations by year: 2026: 28 · 2025: 34 · 2024: 38 · 2023: 40 · 2022: 31 · 2021: 32 · 2020: 48 · 2019: 9
FWCI: 3.98
Type: journal article
Language: en
Open access: gold
doi.org
Licence: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor)
Locations: Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
Europe PMC (PubMed Central) (publishedVersion, cc-by-nc-nd)
PubMed Central (publishedVersion, cc-by-nc-nd)
PubMed (publishedVersion)
DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Funders: Kitzman Foundation
Children's Miracle Network Hospitals
Deutsche Forschungsgemeinschaft
Bundesministerium für Bildung und Forschung
Ministerium für Wissenschaft, Forschung und Kunst Baden-Württemberg
National Institutes of Health (R01 HL-097088, R21 EB-015684, R41 AI-122735)
Niedersächsisches Ministerium für Wissenschaft und Kultur
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
Health Sciences › Medicine › Infectious Diseases › Parvovirus B19 Infection Studies
Keywords: adeno-associated virus vectors, capsid modification, gene therapy vectors, gene delivery, vector targeting, vector safety, parvovirus

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Capsid modifications for targeting and improving the efficacy of AAV vectors

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