Journey to the Center of the Cell: Tracing the Path of AAV Transduction

Dhungel, Bijay P.; Bailey, Charles G.; Rasko, John E.J.

doi:10.1016/j.molmed.2020.09.010

Cited by 122Virus-based gene therapy researchRNA Interference and Gene DeliveryViral Infectious Diseases and Gene Expression in Insects

Journey to the Center of the Cell: Tracing the Path of AAV Transduction

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Dhungel, Bijay P., Bailey, Charles G., Rasko, John E.J.·2021·Trends in Molecular Medicine

DOI 10.1016/j.molmed.2020.09.010

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Abstract

As adeno-associated virus (AAV)-based gene therapies are being increasingly approved for use in humans, it is important that we understand vector-host interactions in detail. With the advances in genome-wide genetic screening tools, a clear picture of AAV-host interactions is beginning to emerge. Understanding these interactions can provide insights into the viral life cycle. Accordingly, novel strategies to circumvent the current limitations of AAV-based vectors may be explored. Here, we summarize our current understanding of the various stages in the journey of the vector from the cell surface to the nucleus and contextualize the roles of recently identified host factors.

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After endosomal escape, rAAV particles either enter the nucleus for transgene expression, or become ubiquitylated then degraded by proteasome.
“After endosomal escape, rAAV particles either enter the nucleus for transgene expression, or become ubiquitylated then degraded by proteasome (84).”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [84]
Endosomal acidification is essential for rAAV escape, and hydroxychloroquine increases endosomal and lysosomal pH, so the effect may not be replicated or consistent with systemic application.
“However, endosomal acidification is essential for rAAV escape (84), and hydroxychloroquine increases endosomal and lysosomal pH (60), this effect may not be replicated or consistent with systemic application.”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [84]

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AAV: An Overview of Unanswered Questions
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Impact of VP1-Specific Protein Sequence Motifs on Adeno-Associated Virus Type 2 Intracellular Trafficking and Nuclear Entry
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Recombinant Adeno-Associated Virus Utilizes Host Cell Nuclear Import Machinery To Enter the Nucleus
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Adeno-Associated Virus Type 2 Binds to a 150-Kilodalton Cell Membrane Glycoprotein
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Adeno-associated virus 2 bound to its cellular receptor AAVR
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Mutagenesis of Adeno-Associated Virus Type 2 Capsid Protein VP1 Uncovers New Roles for Basic Amino Acids in Trafficking and Cell-Specific Transduction
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Engineering the AAV capsid to evade immune responses
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Human Hepatocyte Growth Factor Receptor Is a Cellular Coreceptor for Adeno-Associated Virus Serotype 3
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rAAV2 traffics through both the late and the recycling endosomes in a dose-dependent fashion
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Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction
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Optimization of the Capsid of Recombinant Adeno-Associated Virus 2 (AAV2) Vectors: The Final Threshold?
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Syntaxin 5-Dependent Retrograde Transport to the trans -Golgi Network Is Required for Adeno-Associated Virus Transduction
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Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo
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A Two-hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2 and 8
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Vector Uncoating Limits Adeno-Associated Viral Vector-Mediated Transduction of Human Dendritic Cells and Vector Immunogenicity
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Processing of recombinant AAV genomes occurs in specific nuclear structures that overlap with foci of DNA-damage-response proteins
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Characterization of AAV vector particle stability at the single-capsid level
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Adeno-associated virus (AAV) vectors: Rational design strategies for capsid engineering
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Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR
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Divergent engagements between adeno-associated viruses with their cellular receptor AAVR
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Evidence for pH-Dependent Protease Activity in the Adeno-Associated Virus Capsid
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Super-resolution imaging of nuclear import of adeno-associated virus in live cells
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Adeno-Associated Virus Capsid Proteins May Play a Role in Transcription and Second-Strand Synthesis of Recombinant Genomes
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Parvoviruses Cause Nuclear Envelope Breakdown by Activating Key Enzymes of Mitosis
Porwal, M., Cohen, S., Snoussi, K., Popa-Wagner, R., Anderson, F., Dugot‐Senant, N., Wodrich, H., Dinsart, C., Kleinschmidt, J. A., Panté, N., et al. (2013) · PLoS Pathogens · doi:10.1371/journal.ppat.1003671 · Cited by 68 · see citing works
Adeno-Associated Virus 2 at 2.8 ang
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Mechanistic insights into the enhancement of adeno-associated virus transduction by proteasome inhibitors
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Adeno-associated virus capsid antigen presentation is dependent on endosomal escape
Li, C., He, Y., Nicolson, S., Hirsch, M., Weinberg, M. S., Zhang, P., Kafri, T., & Samulski, R. J. (2013) · Journal of Clinical Investigation · doi:10.1172/jci66611 · Cited by 61 · see citing works
Gene therapy and the adeno‐associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations
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Self-citation
Endocytic processing of adeno-associated virus type 8 vectors for transduction of target cells
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Pooled Screens Identify GPR108 and TM9SF2 as Host Cell Factors Critical for AAV Transduction
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Enhancement of adeno-associated virus-mediated gene therapy using hydroxychloroquine in murine and human tissues
Chandler, L. C., Barnard, A. R., Caddy, S. L., Patrício, M. I., McClements, M. E., Fu, H., Rada, C., MacLaren, R. E., & Xue, K. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.05.012 · Cited by 50 · see citing works
Insight into the mechanism of inhibition of adeno-associated virus by the Mre11/Rad50/Nbs1 complex
Lentz, T. B., & Samulski, R. J. (2015) · Journal of Virology · doi:10.1128/jvi.01990-14 · Cited by 48 · see citing works
Identification and Validation of Small Molecules That Enhance Recombinant Adeno-associated Virus Transduction following High-Throughput Screens
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An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses
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AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6
Denard, J., Rouillon, J., Léger, T., Garcia, C., Lambert, M. P., Griffith, G., Jenny, C., Camadro, J., Garcı́a, L., & Svinartchouk, F. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.08.001 · Cited by 44 · see citing works
Next-generation of targeted AAVP vectors for systemic transgene delivery against cancer
Suwan, K., Yata, T., Waramit, S., Przystal, J. M., Stoneham, C. A., Bentayebi, K., Asavarut, P., Chongchai, A., Pothachareon, P., Lee, K., et al. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1906653116 · Cited by 44 · see citing works
Arsenic Trioxide Stabilizes Accumulations of Adeno-Associated Virus Virions at the Perinuclear Region, Increasing Transduction <i>In Vitro</i> and <i>In Vivo</i>
Mitchell, A. M., Li, C., & Samulski, R. J. (2013) · Journal of Virology · doi:10.1128/jvi.03443-12 · Cited by 41 · see citing works
CD9 promotes adeno-associated virus type 2 infection of mammary carcinoma cells with low cell surface expression of heparan sulphate proteoglycans
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Unique Characteristics of AAV1, 2, and 5 Viral Entry, Intracellular Trafficking, and Nuclear Import Define Transduction Efficiency in HeLa Cells
Keiser, N. W., Yan, Z., Zhang, Y., Lei-Butters, D. C. M., & Engelhardt, J. F. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.044 · Cited by 40 · see citing works
Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction
Sipo, I., Fechner, H., Pinkert, S., Suckau, L., Wang, X., Weger, S., & Poller, W. C. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302987 · Cited by 40 · see citing works
Recombinant Adeno-Associated Virus Utilizes Cell-Specific Infectious Entry Mechanisms
Weinberg, M. S., Nicolson, S. C., Bhatt, A. P., McLendon, M., Li, C., & Samulski, R. J. (2014) · Journal of Virology · doi:10.1128/jvi.01971-14 · Cited by 39 · see citing works
Gene Therapy: It Is Time to Talk about High-Dose AAV
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MicroRNA-Regulated Gene Delivery Systems for Research and Therapeutic Purposes
Dhungel, B. P., Ramlogan‐Steel, C. A., & Steel, J. C. (2018) · Molecules · doi:10.3390/molecules23071500 · Cited by 32 · see citing works
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Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction
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Polarized AAVR expression determines infectivity by AAV gene therapy vectors
Hamilton, B. A., Li, X., Pezzulo, A. A., Alaiwa, M. H. A., & Zabner, J. (2019) · Gene Therapy · doi:10.1038/s41434-019-0078-3 · Cited by 28 · see citing works
Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription
Madigan, V. J., Yuziuk, J. A., Chiarella, A. M., Tyson, T. O., Meganck, R. M., Elmore, Z. C., Tse, L. V., Hathaway, N. A., & Asokan, A. (2019) · PLoS Pathogens · doi:10.1371/journal.ppat.1007988 · Cited by 28 · see citing works
Adeno-Associated Virus VP1u Exhibits Protease Activity
Kurian, J. J., Lakshmanan, R., Chmely, W. M., Hull, J. A., Yu, J. C., Bennett, A. D., McKENNA, R. V., & Agbandje‐McKenna, M. (2019) · Viruses · doi:10.3390/v11050399 · Cited by 26 · see citing works
Promyelocytic Leukemia Protein Is a Cell-Intrinsic Factor Inhibiting Parvovirus DNA Replication
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Effect of bortezomib on the efficacy of AAV9. SERCA2a treatment to preserve cardiac function in a rat pressure-overload model of heart failure
Chaanine, A. H., Nonnenmacher, M., Kohlbrenner, E., Jin, D., Kovacic, J. C., Akar, F. G., Hajjar, R. J., & Weber, T. (2014) · Gene Therapy · doi:10.1038/gt.2014.7 · Cited by 25 · see citing works
Proteasome modulating agents induce rAAV2-mediated transgene expression in human intestinal epithelial cells.
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Seek and destroy: targeted adeno-associated viruses for gene delivery to hepatocellular carcinoma
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Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Markusic, D. M., Nichols, T. C., Merricks, E. P., Palaschak, B., Zolotukhin, I., Marsic, D., Zolotukhin, S., Srivastava, A., & Herzog, R. W. (2017) · Journal of Translational Medicine · doi:10.1186/s12967-017-1200-1 · Cited by 19 · see citing works
Evaluation of the Glypican 3 promoter for transcriptional targeting of hepatocellular carcinoma
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A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes
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Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors
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Tyrosine Mutation in AAV9 Capsid Improves Gene Transfer to the Mouse Lung
Martini, S. V., da Silva, A. L., Pires-Ferreira, D., Rabelo, R., Ornellas, F. M., Gomes, K., Rocco, P. R. M., Petrs‐Silva, H., & Morales, M. M. (2016) · Cellular Physiology and Biochemistry · doi:10.1159/000445646 · Cited by 16 · see citing works
Enhancement of rAAV2-Mediated Transgene Expression in Retina Cells In Vitro and In Vivo by Coadministration of Low-Dose Chemotherapeutic Drugs
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AAV Entry: Filling in the Blanks
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MicroRNA199a-Based Post-transcriptional Detargeting of Gene Vectors for Hepatocellular Carcinoma
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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
“40 For example, rAAV2 binds to heparan sulphate proteoglycan (HSPG), rAAV1, rAAV4, and rAAV5 primarily interact with sialic acid, and rAAV9 interacts with N-linked galactose.”
Background
Adeno-associated virus (AAV) cell entry: structural insights
Meyer, N., & Chapman, M. S. (2021) · Trends in Microbiology · doi:10.1016/j.tim.2021.09.005 · Cited by 139 · see citing works
Continuous evolution of CRISPR-associated transposases for efficient, RNA-programmed gene insertion into the human genome
Witte, I. P., Lampe, G. D., Eitzinger, S., Miller, S. M., Berríos, K. N., McElroy, A., King, R. T., Stringham, O. G., Gelsinger, D., Vo, P., et al. (2025) · Science · doi:10.1126/science.adt5199 · Cited by 76 · see citing works
Extracellular Vesicle–Encapsulated Adeno-Associated Viruses for Therapeutic Gene Delivery to the Heart
Li, X., Salvia, S. L., Liang, Y., Adamiak, M., Kohlbrenner, E., Jeong, D., Chepurko, E., Ceholski, D. K., López-Gordo, E., Yoon, S., et al. (2023) · Circulation · doi:10.1161/circulationaha.122.063759 · Cited by 91 · see citing works
Understanding AAV vector immunogenicity: from particle to patient
Dhungel, B. P., Winburn, I. C., Pereira, C. D. F., Huang, K., Chhabra, A., & Rasko, J. E. J. (2024) · Theranostics · doi:10.7150/thno.89380 · Cited by 90 · see citing works
“…most serotypes can be found in the majority of subjects [14, 31] AAV, adeno-associated virus; NAb, neutralizing antibody; rAAV, recombinant adeno-associated virus AAVs consist of a single-stranded DNA (ssDNA) genome of ~4.7 Kb enclosed by a capsid ~26 nm in diameter ( Figure 1A ) [10, 11].”
BackgroundInfluentialSelf-citation
AAV manufacturing for clinical use: insights on current challenges from the upstream process perspective
Dobrowsky, T., Gianni, D., Pieracci, J., & Suh, J. (2021) · Current Opinion in Biomedical Engineering · doi:10.1016/j.cobme.2021.100353 · Cited by 90 · see citing works
Artificial miRNAs as therapeutic tools: Challenges and opportunities
Kotowska‐Zimmer, A., Pewińska, M., & Olejniczak, M. (2021) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.1640 · Cited by 80 · see citing works
“A large variety of capsids have been developed to prevent neutralization by antibodies targeting specific capsids, which improves the in vivo transduction efficiency (Dhungel et al., 2020; Wu et al., 2006).”
Background
Adeno-associated viruses for gene therapy – clinical implications and liver-related complications, a guide for hepatologists
Mücke, M. M., Fong, S., Foster, G. R., Lillicrap, D., Miesbach, W., & Zeuzem, S. (2023) · Journal of Hepatology · doi:10.1016/j.jhep.2023.10.029 · Cited by 71 · see citing works
CRISPR FISHer enables high-sensitivity imaging of nonrepetitive DNA in living cells through phase separation-mediated signal amplification
Lyu, X.-Y., Deng, Y., Huang, X., Li, Z., Fang, G.-Q., Dong, Y., Wang, F.-L., Kang, W., Shen, E.-Z., & Song, C. (2022) · Cell Research · doi:10.1038/s41422-022-00712-z · Cited by 69 · see citing works
“Adeno-associated virus (AAV) is an attractive vector in human gene therapy because it is a nonpathogenic parvovirus.(32) Double-strand (ds) AAV DNA, generated from replication of its single-strand DNA, enables CRISPR FISHer targeting (Fig.”
AAV-mediated gene therapy: Advancing cardiovascular disease treatment
Zhang, H., Qi, Z., Huang, B., Wang, Y., & Wang, X. (2022) · Frontiers in Cardiovascular Medicine · doi:10.3389/fcvm.2022.952755 · Cited by 67 · 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
“On the other hand, the efforts to identify receptors for novel capsids have significant theoretical meanings, as AAV transduction is a complicated and yet-to-be-elucidated process that involves multiple steps that can affect transgene expression [89].”
Background
Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies
Chu, W. S., & Ng, J. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.658038 · Cited by 59 · see citing works
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review
Wang, J.-H., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.10.017 · Cited by 48 · 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
Structural basis for the neurotropic AAV9 and the engineered AAVPHP.eB recognition with cellular receptors
Xu, G., Zhang, R., Li, H., Yin, K., Ma, X., & Lou, Z. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.05.009 · Cited by 42 · see citing works
“Recent study elucidated that AAVR interacting VR-I also participates in AAV9 galactose binding and might have been selected during evolution to ensure the virus internalization;(15) this is in line with the role of AAVR facilitating intracellular trafficking of the virus.(9,28)”
Optimization of AAV6 transduction enhances site-specific genome editing of primary human lymphocytes
Rogers, G. L., Huang, C., Clark, R. D. E., Seclén, E., Chen, H.-Y., & Cannon, P. M. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.09.003 · Cited by 37 · see citing works
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
Marcó, S., Haurigot, V., Jaén, M. L., Ribera, A., Sánchez, V., Molas, M., Garcia, M., León, X., Roca, C., Sánchez, X., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.09.017 · Cited by 36 · see citing works
“Whether the difference in VGCN reported in short- versus long-term studies reflects non-functional vector genomes that were not taken up by cells and are cleared with time, stabilization of only an initial fraction of the vector genomes in the nucleus, or turnover of some of the targeted cells remains to be determined…”
Background
The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution
Kachanov, A., Kostyusheva, A., Brezgin, S., Karandashov, I., Ponomareva, N. I., Tikhonov, A., Lukashev, A. N., Pokrovsky, V. S., Zamyatnin, A. A., Parodi, A., et al. (2024) · Medicinal Research Reviews · doi:10.1002/med.22036 · Cited by 38 · see citing works
AAV genome modification for efficient AAV production
Asaad, W., Volos, P., Maksimov, D. O., Khavina, E. M., Deviatkin, A. A., Mityaeva, O. N., & Volchkov, P. (2023) · Heliyon · doi:10.1016/j.heliyon.2023.e15071 · Cited by 29 · see citing works
Engineering challenges and translational opportunities in emerging gene delivery platforms
Ma, Y., Dong, S., Wu, A., Jeong, S. D., Lee, A. S., Jiang, W., & Kim, B. Y. S. (2026) · Nature Biomedical Engineering · doi:10.1038/s41551-026-01643-5 · Cited by 30 · see citing works
“AAVs outperform non-viral platforms due to capsid-mediated, pH-dependent membrane perforation, aided by the VP1 phospholipase A2 domain 88–90 .”
PCR-Based Analytical Methods for Quantification and Quality Control of Recombinant Adeno-Associated Viral Vector Preparations
Shmidt, A. A., & Egorova, T. V. (2021) · Pharmaceuticals · doi:10.3390/ph15010023 · Cited by 26 · see citing works
Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy
Purdy, R., John, M., Bray, A., Clare, A. J., Copland, D. A., Chan, Y. K., Henderson, R. H., Nerinckx, F., Leroy, B. P., Yang, P., et al. (2025) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2025.101354 · Cited by 24 · see citing works
“Detail on viral entry is provided elsewhere (Dhungel et al., 2021; J.-H. Wang et al., 2024).”
Dysregulation of miRNAs in DLBCL: Causative Factor for Pathogenesis, Diagnosis and Prognosis
Alsaadi, M., Khan, M. Y., Dalhat, M. H., Bahashwan, S., Khan, M. U., Albar, A. H., Almehdar, H. A., & Qadri, I. (2021) · Diagnostics · doi:10.3390/diagnostics11101739 · Cited by 23 · see citing works
“Viral delivery systems a remarkably efficient system, however, these may elicit immunogenic responses which halt their effectiveness [26].”
Background
The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy
Puzzo, F., & Kay, M. A. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.041 · Cited by 24 · see citing works
“75,87 This phenomenon has been hypothesized to result from species-specific intracellular processing mechanisms 88 or epigenetic regulation of the AAV genome unique to each species.”
Targeting the somatosensory system with AAV9 and AAV2retro viral vectors
Skorput, A. G. J., Gore, R., Schorn, R. E., Riedl, M. S., de Velasco, E. M. F., Hadlich, B., Kitto, K. F., Fairbanks, C. A., & Vulchanova, L. (2022) · PLoS ONE · doi:10.1371/journal.pone.0264938 · Cited by 22 · see citing works
“AAV9 and AAV2 (the parent serotype of AAV2retro) bind to different primary and secondary receptors on the cell surface [32].”
Background
Correction of LAMP3-associated salivary gland hypofunction by aquaporin gene therapy
Nakamura, H., Tanaka, T., Zheng, C., Afione, S. A., Warner, B. M., Noguchi, M., Atsumi, T., & Chiorini, J. A. (2022) · Scientific Reports · doi:10.1038/s41598-022-21374-2 · Cited by 22 · see citing works
Retina-directed gene therapy: Achievements and remaining challenges
Biber, J., Gandor, C. L. A., Bećirović, E., & Michalakis, S. (2025) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2025.108862 · Cited by 24 · see citing works
An alternate receptor for adeno-associated viruses
Dhungel, B. P., Xu, H., Nagarajah, R., Vitale, J. N., Wong, A. C. H., Gokal, D., Feng, Y., Tabar, M. S., Metierre, C., Parsania, C., et al. (2025) · Cell · doi:10.1016/j.cell.2025.06.026 · Cited by 21 · see citing works
Self-citation
A robust and efficient alluvial filtration method for the clarification of adeno-associated viruses from crude cell lysates
Meierrieks, F., Pickl, A., & Wolff, M. W. (2023) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2023.03.010 · Cited by 23 · see citing works
Chemical approaches to probe and engineer AAV vectors
Pham, Q. T., Glicksman, J., & Chatterjee, A. (2024) · Nanoscale · doi:10.1039/d4nr01300j · Cited by 23 · see citing works
Adeno-associated viral tools to trace neural development and connectivity across amphibians
Jaeger, E. C. B., Vijatovic, D., Deryckere, A., Zorin, N., Nguyen, A. L., Ivanian, G., Woych, J., Arnold, R. C., Gurrola, A. O., Shvartsman, A., et al. (2024) · Developmental Cell · doi:10.1016/j.devcel.2024.10.025 · Cited by 21 · see citing works
Aptamer-based applications in delivering cancer gene therapies and beyond: state of the art and the missing links to clinical translation
Coppola, G., Cennamo, F., Ciccone, G., Ibba, M. L., Ruscio, A. D., Vito, A. D., Esposito, C. L., & Catuogno, S. (2025) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2025.115639 · Cited by 17 · see citing works
“Beyond Ad5, other commonly used types of viruses are the adeno-associated (AAV), a class of non-pathological viruses characterized by a broad tropism toward different cells thanks to their recognition of glycans, glycoconjugates, or sialic acid [97].”
Biophysical characterization of adeno-associated virus capsid through the viral transduction life cycle
Xiang, Y. S., & Hao, G. G. (2023) · Journal of Genetic Engineering and Biotechnology · doi:10.1186/s43141-023-00518-5 · Cited by 17 · see citing works
“These include cell surface receptor recognition, endosomal trafficking and escape, nuclear entry, genome uncoating, viral replication and assembly, and immune neutralization and proteosome degradation [16, 17].”
Background
Increasing Gene Editing Efficiency via CRISPR/Cas9- or Cas12a-Mediated Knock-In in Primary Human T Cells
Kruglova, N. A., & Shepelev, M. V. (2024) · Biomedicines · doi:10.3390/biomedicines12010119 · Cited by 18 · see citing works
“Purification and activation methods should convert T cells into a state amenable to editing, which includes the efficient delivery and nuclear translocation of the CRISPR/Cas components, as well as rapid progression through the cell cycle required for HDR. Purification, activation, media…”
Method
Changes in Myonuclear Number During Postnatal Growth – Implications for AAV Gene Therapy for Muscular Dystrophy
Morgan, J. E., & Francesco Muntoni, A. (2021) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-210683 · Cited by 16 · see citing works
“AAV enters nuclei where it usually remains episomal (reviewed [40]); integration into the host genome is minimal.”
Background
The Expression and Function of the Small Nonstructural Proteins of Adeno-Associated Viruses (AAVs)
Küz, Ç. A., McFarlin, S., & Qiu, J. (2024) · Viruses · doi:10.3390/v16081215 · Cited by 15 · see citing works
“During infection, a conformational change in the capsid is necessary, resulting in the protrusion of the N-termini of VP1u, leading to endosomal escape via PLA2 activity, and of the VP2u out of the intact capsid, routing the capsid to the nucleus by interacting with the NLSs [37,80].”
Background
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
“In addition, the degree of structural freedom of the VP1/VP2 unique region and the fivefold symmetry axis channel region of the complete particle is lower than that of the empty particle [76].”
Background
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
“…and sorting, cytoplasmic escape, nuclear import, genome release, and transgene expression (Figure 2) [20]. successive cellular events, including attachment, entry, internalization and sorting, cytoplasmic escape, nuclear import, genome release, and transgene expression (Figure 2) [20].”
Influential
Biomaterial-Based Nucleic Acid Delivery Systems for In Situ Tissue Engineering and Regenerative Medicine
Wu, Q.-X., de Isla, N., & Zhang, L. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26157384 · Cited by 14 · see citing works
Gene therapy – are we ready now?
Kaczmarek, R. (2022) · Haemophilia · doi:10.1111/hae.14530 · Cited by 13 · see citing works
Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing
Moyo, B., Brown, L. B. C., Khondaker, I., & Bao, G. (2025) · Biomaterials · doi:10.1016/j.biomaterials.2025.123314 · Cited by 13 · see citing works
Structural characterization of the porcine adeno‐associated virus Po1 capsid protein binding to the nuclear trafficking protein importin alpha
Hoad, M., Roby, J. A., & Forwood, J. K. (2021) · FEBS Letters · doi:10.1002/1873-3468.14209 · Cited by 10 · see citing works
Unveiling the secrets of adeno-associated virus: novel high-throughput approaches for the quantification of multiple serotypes
Meierrieks, F., Kour, A., Pätz, M., Pflanz, K. B., Wolff, M. W., & Pickl, A. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101118 · Cited by 10 · see citing works
Biodistribution of Adeno-Associated Virus Serotype 5 Viral Vectors Following Intrathecal Injection
Pflepsen, K. R., Peterson, C. D., Kitto, K. F., Riedl, M. S., McIvor, R. S., Wilcox, G. L., Vulchanova, L., & Fairbanks, C. A. (2021) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.1c00252 · Cited by 10 · see citing works
Manufacturing and Quality of Advanced Therapeutics: Cellular Therapies, CAR-T and Gene Therapies
van den Bos, C., Keefe, R., Keville, M., & McCaman, M. T. (2023) · Cell engineering · doi:10.1007/978-3-031-45669-5_15 · Cited by 9 · see citing works
Identification of SLC35A1 as an essential host factor for the transduction of multi-serotype recombinant adeno-associated virus (AAV) vectors
Zhang, X., Hao, S., Feng, Z., Ning, K., Küz, Ç. A., McFarlin, S., Richart, D., Cheng, F., Zhang-Chen, A., McFarlane, R., et al. (2024) · mBio · doi:10.1128/mbio.03268-24 · Cited by 8 · see citing works
Targeted Delivery of Chimeric Antigen Receptor into T Cells via CRISPR-Mediated Homology-Directed Repair with a Dual-AAV6 Transduction System
Moço, P. D., Farnós, O., Sharon, D. M., & Kamen, A. (2023) · Current Issues in Molecular Biology · doi:10.3390/cimb45100486 · Cited by 8 · see citing works
“In most cases, the AAV genome remains in an episomal state, which confers low toxicity and allows for the expression of transgenes for long periods of time in non-dividing cells [8].”
Background
Generation of scalable cancer models by combining AAV-intron-trap, CRISPR/Cas9, and inducible Cre-recombinase
Boddu, P. C., Gupta, A. K., Kim, J., Neugebauer, K. M., Waldman, T. A., & Pillai, M. M. (2021) · Communications Biology · doi:10.1038/s42003-021-02690-1 · Cited by 8 · see citing works
Myospreader improves gene editing in skeletal muscle by myonuclear propagation
Poukalov, K., Valero, M. C., Muscato, D. R., Adams, L. M., Chun, H., Lee, Y. I., Andrade, N. S., Zeier, Z. R., Sweeney, H. L., & Wang, E. T. (2024) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2321438121 · Cited by 7 · see citing works
Neuroglobin overexpression in cerebellar neurons of Harlequin mice improves mitochondrial homeostasis and reduces ataxic behavior
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
“41 So, the treatment of Hq mice at the age of 2 months could be too late for ef ﬁ ciently preventing neuronal cell loss.”
Development of an adipose-tropic AAV capsid ablating liver tropism
Huang, W., Bates, R., Appana, B., Mohammed, T., & Cao, L. (2024) · iScience · doi:10.1016/j.isci.2024.110930 · Cited by 7 · see citing works
Toxicologic Neuropathology of Novel Biotherapeutics
Bangari, D. S., Lanigan, L. G., Cramer, S. D., Grieves, J. L., Meisner, R., Rogers, A. B., Galbreath, E. J., & Bolon, B. N. (2023) · Toxicologic Pathology · doi:10.1177/01926233241230542 · Cited by 7 · see citing works
“42,46,112 Certain AAV serotypes and differences in expression cassette engineering (such as a tissue-or cellular-directed promoter) have an enhanced affinity for neural expression.”
Background
Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors
Rostami, M. R., Leopold, P. L., Vasquez, J. M., Rougvie, M. D. M., Al-Shakaki, A., Hssain, A. A., Robay, A., Hackett, N. R., Mezey, J. G., & Crystal, R. G. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101136 · Cited by 5 · see citing works
Genome-wide CRISPR screenings identified SMCHD1 as a host-restricting factor for AAV transduction
Wang, C., Liu, Y., Xiong, J., Xie, K., Wang, T., Hu, Y., Fu, H., Zhang, B., Huang, X., Bao, H., et al. (2024) · PLoS Pathogens · doi:10.1371/journal.ppat.1012344 · Cited by 6 · see citing works
“Due to the lack of naturally encoded viral proteins and helper viruses, each step of AAV transduction heavily relied on the exploitation of host cellular machinery [12].”
Background
Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy
Meng, J., Moore, M., Counsell, J. R., Francesco Muntoni, A., Popplewell, L. J., & Morgan, J. E. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.04.015 · Cited by 6 · see citing works
Adeno-Associated Virus-like Particles’ Response to pH Changes as Revealed by nES-DMA
Zoratto, S., Heuser, T., Friedbacher, G., Pletzenauer, R., Graninger, M., Marchetti‐Deschmann, M., & Weiss, V. U. (2023) · Viruses · doi:10.3390/v15061361 · Cited by 6 · see citing works
“Other viruses instead—AAV8 included—need to survive and accomplish endosomal escape intact in order to complete the infection [38,39].”
Background
In vivo labeling and quantitative imaging of neuronal populations using MRI
Li, S., Xu, X., Li, C., Xu, Z., Wu, K., Ye, Q., Zhang, Y., Jiang, X., Cang, C., Tian, C., et al. (2023) · NeuroImage · doi:10.1016/j.neuroimage.2023.120374 · Cited by 6 · see citing works
“This viral vector serves as an effective tool for expressing foreign genes (Dhungel et al., 2021; Koyuncu et al., 2013; Polinski et al., 2015; Tervo et al., 2016).”
Stem cell and gene therapies for leukodystrophies
Lin, W., Zhang, M., Zheng, S., & Lian, Q. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101527 · Cited by 6 · see citing works
“36–38 AAV-mediated GRT aims to restore normal gene function by delivering functional copies of defective genes for monogenic leukodystrophies.”
The significance of triple-capsid-mutant AAV8 for treatment of Sanfilippo Syndrome Type B
Ashby, F. J., & Heldermon, C. D. (2022) · Archives of Stem Cell and Therapy · doi:10.46439/stemcell.3.013 · Cited by 4 · see citing works
The vectors went in two-by-two: Transduction efficiency and tolerability of dual and triple rAAV vector delivery following intravitreal injection for genome-editing applications
Fehrman, R. L., Chern, K. J., Stoltz, K. P., & Lipinski, D. M. (2024) · Experimental Eye Research · doi:10.1016/j.exer.2024.110223 · Cited by 5 · see citing works
A custom-made AAV1 variant (AAV1-T593K) enables efficient transduction of Japanese quail neurons in vitro and in vivo
Zoabi, S., Andreyanov, M., Heinrich, R., Ron, S., Carmi, I., Gutfreund, Y., & Berlin, S. (2023) · Communications Biology · doi:10.1038/s42003-023-04712-6 · Cited by 5 · see citing works
Investigation of Lysosome‐Associated Membrane Protein 3 Highlighting the Role of Lysosome in Pathophysiology and Treatment of Sjögren Disease
Nakamura, H., Tanaka, T., Noguchi, M., Atsumi, T., Warner, B. M., & Chiorini, J. A. (2025) · Arthritis & Rheumatology · doi:10.1002/art.43347 · Cited by 4 · see citing works
“85 Disease processes that affect intracellular traf ﬁ cking can block the ef ﬁ ciency of AAV transduction.”
Skeleton keys and Trojan horses: a review of therapeutic delivery to the brain
Stoub, R. E., & Bailus, B. J. (2025) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2025.1674333 · Cited by 4 · see citing works
Identification of CD164 as an essential entry receptor for divergent adeno-associated viruses
Zhang, X., Richart, D., McFarlin, S., Cheng, F., Park, S. Y., Zhang-Chen, A., McFarlane, R., Xiao, C., Yan, Z., & Jianming, Q. (2026) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2525865123 · Cited by 4 · see citing works
Engineering immune cells for next-generation therapies
Han, M., Lou, J., Pan, M., Xu, S., Jiang, X., & Jing, W. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.115012 · Cited by 4 · see citing works
Hippocampal CA2 neurons disproportionately express AAV-delivered genetic cargoPreprint
Alexander, G. M., He, B., Leikvoll, A., Jones, S., Wine, R. N., Kara, P., Martin, N. P., & Dudek, S. M. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.11.27.625768 · Cited by 5 · see citing works
Evaluation of the SH-SY5Y cell line as an in vitro model for potency testing of a neuropeptide-expressing AAV vector
Zanker, J., Hüser, D., Savy, A., Lázaro-Petri, S., Hammer, E.-M., Schwarzer, C., & Heilbronn, R. (2023) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2023.1280556 · Cited by 3 · see citing works
The hepatic Odyssey of Adeno-associated virus: from gene delivery to long-term outcomes
Simini, G., & Batty, P. A. (2025) · Journal of Thrombosis and Haemostasis · doi:10.1016/j.jtha.2025.08.028 · Cited by 3 · see citing works
Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression
Fong, S., Swystun, L. L., Batty, P. A., & Lillicrap, D. (2026) · Therapeutic Advances in Hematology · doi:10.1177/20406207251406537 · Cited by 3 · see citing works
Recent Advances in Designing Adeno-Associated Virus-Based Vaccines Against Viral Infections
Mnyandu, N., Jacobs, R., Arbuthnot, P. B., & Maepa, M. B. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16111360 · Cited by 3 · see citing works
“This is followed by viral assembly and release (Figure 2) [18]. followed by rep protein synthesis (11), viral assembly, and release (12) (Created in BioRender.com).”
Development of a Beta Cell-Specific Expression Control Element for Recombinant Adeno-Associated Virus
Chai, S., Kim, Y., Galivo, F., Dorrell, C., Wakefield, L., Posey, J., Ackermann, A. M., Kaestner, K. H., Hebrok, M., & Grompe, M. (2022) · Human Gene Therapy · doi:10.1089/hum.2021.219 · Cited by 3 · see citing works
CRISPR screen for rAAV production implicates genes associated with infectionPreprint
O'Driscoll, E., Arora, S., Lang, J. F., Davidson, B. L., & Shalem, O. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.09.17.613356 · Cited by 3 · see citing works
CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production
O'Driscoll, E., Arora, S., Lang, J. F., Davidson, B. L., & Shalem, O. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101408 · Cited by 5 · see citing works
Serpin B9‐Insensitive Granzyme B Mutant Delivered by Engineered Capsid AAV Vectors Demonstrates Selective Killing of EGFR‐Positive Cancer Cells
Dogbey, D. M., Naran, K., & Barth, S. (2025) · Advances in Cell and Gene Therapy · doi:10.1155/acg2/8881294 · Cited by 2 · see citing works
“Herein, we demonstrated a shift from clathrin and caveolin-mediated cell entry by wild type AAVs [43] to receptor-dependent transduction by the engineered vectors.”
The Cold Case Files of rAAV Capsid Influence on Transduction: New Leads
Powell, S. (2025) · Viruses · doi:10.3390/v17111476 · Cited by 2 · see citing works
“An overview of rAAV transduction. rAAV transduction steps have been thoroughly reviewed elsewhere [18] but is briefly depicted above in numerical order. rAAV binds to cellular attachment factors as well as primary and secondary receptors (step 1) which enable cellular internalization in step 2.…”
Epilepsy Treatment through Genetic Intervention of GABAergic Interneurons: Promises and Challenges
Podgorny, O. V., Petukhova, E. O., & Belousov, V. V. (2025) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.5c00747 · Cited by 2 · see citing works
Synthetic serum markers enable noninvasive monitoring of gene expression in primate brains
Lee, S., Romac, M. D., Watanabe, S., Chernov, M. M., Li, H., Raisley, E. K., Rothenhoefer, K. M., Dahlquist, Z., Szablowski, J. O., & Costa, V. D. (2026) · Neuron · doi:10.1016/j.neuron.2026.01.003 · Cited by 2 · see citing works
Tissue‐specific gene delivery approaches
Nasr, S. S., Cheema, Y., Stern, A., Tabah, O., Poore, S., & Duncan, G. A. (2026) · Bioengineering & Translational Medicine · doi:10.1002/btm2.70125 · Cited by 3 · see citing works
Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development
Munir, M., Butcher, N. J., Werder, R. B., Ranganathan, S., Burow, R., Venables, A., & Kaminskas, L. M. (2026) · Nanomedicine · doi:10.1080/17435889.2026.2640157 · Cited by 2 · see citing works
“Due to their smaller size, AAV then have a better capacity to enter the nucleus without requiring initial capsid disintegration [84].”
Hyperthermia Enhances Adeno-Associated Virus Vector Transduction Efficiency in Melanoma Cells
Bieńkowska, A., Stelmaszczyk‐Emmel, A., Demkow, U. A., & Małecki, M. (2023) · Current Issues in Molecular Biology · doi:10.3390/cimb45100537 · Cited by 2 · see citing works
“Interestingly, HSP70 supports clathrin-dependent endocytosis [62], which is known to be one of the ways that rAAV enters cells [63].”
Background
Gene therapy targeting the blood-brain barrier
Körbelin, J., Pereira, A. A., & Schwaninger, M. (2024) · Vitamins and hormones · doi:10.1016/bs.vh.2024.03.001 · Cited by 2 · see citing works
Endothelial-to-mesenchymal transition enhances permissiveness to AAV vectors in cardiac endothelial cells
Volf, N., Vuerich, R., Colliva, A., Volpe, M. C., Marengon, M., Zentilin, L., Giacca, M., Ring, N., Vodret, S., Braga, L., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.08.014 · Cited by 2 · see citing works
“…gene therapy, HTS has been used to trace the path of AAV vectors inside mammalian cells and discover major determinants of AAV transduction 15,16 This is the ﬁ rst HTS aiming at identifying FDA-approved drugs able to increase the ef ﬁ ciency of transduction using AAV vectors, specifically…”
AAV-microutrophin gene therapy confers durable cardioprotection against pharmacologic and exercise-induced injury in the mdx mouse
Kasden, C. M., Corzo, M. P., Greer, C. D., Singh, T., Reyes, R., Castillo, A., Arany, Z. P., Kozyak, B. W., & Stedman, H. H. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.07.013 · Cited by 2 · see citing works
Artificial miRNAs are potential gene therapy tools, especially for incurable monogenic disordersPreprint
Dowaidar, M. (2021) · doi:10.31219/osf.io/d5rnm · Cited by 2 · see citing works
Self-silencing adenovirus enables precise infectious titration of recombinant adeno-associated viral vectors
Fustinoni, C., Liu, X., Chen, Z., Cawood, R., Valenti, W., Patrício, M. I., & Su, W. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101492 · Cited by 1 · see citing works
“13,15”
Enhanced AAV production via rational design of a novel pHelper vector integrated with HSV-1 helper genes
Ren, H., Nie, J., Song, Z., Mo, W., Yang, Y., & Bai, Z. (2026) · Synthetic and Systems Biotechnology · doi:10.1016/j.synbio.2025.12.018 · Cited by 1 · see citing works
“As biosynthesis of AAV is a complex process, we speculate that additional steps such as genome processing, genome encapsidation, and virion release may affect AAV production as well [16,42,43].”
New Approaches to Cardiac Gene Therapy Delivery
Veera, S., Lin, N., Mavropoulos, S. A., & Ishikawa, K. (2026) · Canadian Journal of Cardiology · doi:10.1016/j.cjca.2026.02.031 · Cited by 1 · see citing works
STARR-CRAAVT: A platform to identify cell type-specific regulatory elements for next-generation gene therapy
Becker, R., Choudhury, P., Oti, M., Fouani, Y., Strobel, B., Ketterer, S., Rumpel, S., Park, J., Kreuz, S., Maier, U., et al. (2026) · iScience · doi:10.1016/j.isci.2026.115469 · Cited by 1 · see citing works
“62,63 Indeed, a study comparing different plasmid-and lentivirus-based MPRA approaches found that enhancer activity can be influenced by relative positioning of the enhancer but also by other screening backbone design differences.”
Deep-sequencing-guided library screening and profiling of AAV capsids in the primate retina
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“Through cross-species research, we can also identify receptors for novel capsids [83, 84], which helps elucidate the mechanism of AAV transduction and transgene expression [85].”
The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness
Zhou, J. (2025) · American Journal of Stem Cells · doi:10.62347/fesn5062 · Cited by 1 · see citing works
“Once released, single-stranded DNA is converted to double-stranded DNA, resulting in the expression of the recombinant transgene [37-39].”
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Influential

Pharmacological and Biophysical Dissections of the Trafficking and Infection of Adeno-Associated Virus (AAV) In Vitro and In Vivo
Xiao, P. (2019) · Carolina Digital Repository (University of North Carolina at Chapel Hill) · doi:10.17615/rn9x-xq74
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Host Factors Affecting AAV Transduction
Madigan, V. J. (2022) · Carolina Digital Repository (University of North Carolina at Chapel Hill) · doi:10.17615/6y70-9a48

Authors: Dhungel, B. P., Bailey, C. G., & Rasko, J. E. J.
Affiliations: The University of Sydney (AU)
Corresponding author: Rasko, John E.J.
ORCID: Dhungel, Bijay P.
Bailey, Charles G.
Rasko, John E.J.
Venue: Trends in Molecular Medicine
Publisher: Elsevier BV
Published: 2021-02
Volume: 27
Issue: 2
Pages: 172-184
ISSN: 1471-4914 (ISSN-L)
DOI: 10.1016/j.molmed.2020.09.010
OpenAlex: W3092811898
Cited by: 122
References: 139
Citations by year: 2026: 32 · 2025: 29 · 2024: 21 · 2023: 17 · 2022: 9 · 2021: 14
FWCI: 1.43
Type: journal article
Language: en
Open access: bronze
doi.org
Licence: www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
doi.org/10.15223/policy-017 (stm-asf)
doi.org/10.15223/policy-037 (stm-asf)
doi.org/10.15223/policy-012 (stm-asf)
doi.org/10.15223/policy-029 (stm-asf)
doi.org/10.15223/policy-004 (stm-asf)
Locations: Trends in Molecular Medicine (publishedVersion)
PubMed (publishedVersion)
Funders: NSW Health
National Health and Medical Research Council
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 › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
Keywords: adeno-associated virus, adeno-associated virus vectors, gene therapy, vector-host interactions, viral life cycle, host factors, nuclear translocation, viral transduction, genome-wide screen
MeSH terms: Host Microbial Interactions, Dependovirus, Animals, Biological Transport, Genetic Vectors, Humans, Transduction, Genetic, Genetic Therapy, Gene Transfer Techniques, Cell Tracking

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Journey to the Center of the Cell: Tracing the Path of AAV Transduction

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