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Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?

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Improving Molecular Therapy in the Kidney

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Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

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Structures and Functions of Parvovirus Capsids and the Process of Cell Infection

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Targeted Gene Therapy for the Treatment of Heart Failure

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Translating CRISPR-Cas Therapeutics: Approaches and Challenges

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Process Improvement of Adeno-Associated Virus Production

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A Versatile Adeno-Associated Virus Vector Producer Cell Line Method for Scalable Vector Production of Different Serotypes

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Cardiac-targeted delivery of regulatory RNA molecules and genes for the treatment of heart failure

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A Robust and All-Inclusive Pipeline for Shuffling of Adeno-Associated Viruses

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Efficacy of Codelivery of Dual AAV2/5 Vectors in the Murine Retina and Hippocampus

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The Nontoxic Cell Cycle Modulator Indirubin Augments Transduction of Adeno-Associated Viral Vectors and Zinc-Finger Nuclease-Mediated Gene Targeting

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Recombinant Adeno-Associated Virus Utilizes Cell-Specific Infectious Entry Mechanisms

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Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation

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The 2.8 Å Electron Microscopy Structure of Adeno-Associated Virus-DJ Bound by a Heparinoid Pentasaccharide

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Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy

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Gene regulation technologies for gene and cell therapy

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Present and Future of Adeno Associated Virus Based Gene Therapy Approaches

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High-Throughput Dissection of AAV–Host Interactions: The Fast and the Curious

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

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Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches

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Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies

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Broadening the Heterologous Cross-Neutralizing Antibody Inducing Ability of Porcine Reproductive and Respiratory Syndrome Virus by Breeding the GP4 or M genes

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Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

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Enhancing the Utility of Adeno-Associated Virus Gene Transfer through Inducible Tissue-Specific Expression

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Recent Advances in RNA Interference Therapeutics for CNS Diseases

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Reducing the Risk of Adeno-Associated Virus (AAV) Vector Mobilization with AAV Type 5 Vectors

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Phase-Separated Liposomes Enhance the Efficiency of Macromolecular Delivery to the Cellular Cytoplasm

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Computationally guided AAV engineering for enhanced gene delivery

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Gene Transfer Therapy for Neurodevelopmental Disorders

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Gene and cell therapy based treatment strategies for inflammatory bowel diseases

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Adeno-associated Virus Vectors Serotype 2 Induce Prolonged Proliferation of Capsid-Specific CD8+ T Cells in Mice

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Recombinant adeno-associated Virus: Clinical Application and Development As a gene-therapy Vector

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Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies

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Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion

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Adeno-associated Virus (AAV) Capsid Chimeras with Enhanced Infectivity Reveal a Core Element in the AAV Genome Critical for both Cell Transduction and Capsid Assembly

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AAV Vector-Mediated Antibody Delivery (A-MAD) in the Central Nervous System

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Development and Validation of Novel AAV2 Random Libraries Displaying Peptides of Diverse Lengths and at Diverse Capsid Positions

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Lung Tissue Delivery of Virus-Like Particles Mediated by Macrolide Antibiotics

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AAV8 virions hijack serum proteins to increase hepatocyte binding for transduction enhancement

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

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AAV-6 mediated efficient transduction of mouse lower airways

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Chemical approaches to probe and engineer AAV vectors

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Optimized AAV rh. 10 vectors that partially evade neutralizing antibodies during hepatic gene transfer

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Single-Point Mutations in Qβ Virus-like Particles Change Binding to Cells

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Viral vector-mediated gene transfer for CNS disease

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Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis

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Prospects for gene therapy in inflammatory arthritis

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Recent development of AAV-based gene therapies for inner ear disorders

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Gene Therapy for Heart Failure: Where Do We Stand?

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An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8+ T Cells

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Gene therapy of the central nervous system: General considerations on viral vectors for gene transfer into the brain

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A NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (AAV)-BASED RANDOM PEPTIDE DISPLAY LIBRARY SYSTEM: INFECTION-DEFECTIVE AAV1.9-3 AS A NOVEL DETARGETED PLATFORM FOR VECTOR EVOLUTION

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Single Tyrosine Mutation in AAV8 and AAV9 Capsids Is Insufficient to Enhance Gene Delivery to Skeletal Muscle and Heart

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Erratum to: Phase-Separated Liposomes Enhance the Efficiency of Macromolecular Delivery to the Cellular Cytoplasm

Imam, Z. I., Kenyon, L. E., Ashby, G., Nagib, F., Mendicino, M., Zhao, C., Gadok, A. K., & Stachowiak, J. C. (2017) · Cellular and Molecular Bioengineering · doi:10.1007/s12195-017-0491-x · Cited by 18

Protein Grafting Techniques: From Peptide Epitopes to Lasso‐Grafted Neobiologics

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Combinatorial Approaches for the Identification of Brain Drug Delivery Targets

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

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Intranasal application of adeno-associated viruses: a systematic review

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Sites in the AAV5 capsid tolerant to deletions and tandem duplications

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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 18

Impact of the Assembly-Activating Protein on Molecular Evolution of Synthetic Adeno-Associated Virus Capsids

Herrmann, A.-K., Große, S., Börner, K., Krämer, C., Wiedtke, E., Gunkel, M., & Grimm, D. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.085 · Cited by 17

Breaking the sound barrier: Towards next-generation AAV vectors for gene therapy of hearing disorders

Fakhiri, J., Landegger, L. D., & Grimm, D. (2020) · Hearing Research · doi:10.1016/j.heares.2020.108092 · Cited by 15

Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein

Maurer, A. C., Diaz, A. K. C., & Vandenberghe, L. H. (2019) · Journal of Virology · doi:10.1128/jvi.02013-18 · Cited by 16

Selective tropism of the recombinant adeno‐associated virus 9 serotype for rat cardiac tissue

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Advanced biomanufacturing and evaluation of adeno-associated virus

Chen, K., Kim, S., Yang, S., Varadkar, T., Zhou, Z. “., Zhang, J., Zhou, L., & Liu, M. (2024) · Journal of Biological Engineering · doi:10.1186/s13036-024-00409-4 · Cited by 15

Novel Lung Tropic Adeno-Associated Virus Capsids for Therapeutic Gene Delivery

Carneiro, A. D., Lee, H., Lin, L. W., van Haasteren, J., & Schaffer, D. V. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.169 · Cited by 13

Altering Tropism of rAAV by Directed Evolution

Marsic, D., & Zolotukhin, S. (2015) · Methods in molecular biology · doi:10.1007/978-1-4939-3271-9_11 · Cited by 12

AAV2-antiVEGFscFv gene therapy for retinal neovascularization

Han, N., Xu, X., Liu, Y., & Luo, G. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101145 · Cited by 10

A facile chemical strategy to synthesize precise AAV-protein conjugates for targeted gene delivery

Pham, Q. T., Glicksman, J., Han, B., Shahraeini, S. S., Jewel, D., Loynd, C., Roy, S. J. S., & Chatterjee, A. (2025) · Molecular Therapy Oncology · doi:10.1016/j.omton.2025.201040 · Cited by 10

Microcontact printing for co-patterning cells and viruses for spatially controlled substrate-mediated gene delivery

McConnell, K. I., Slater, J. H., Han, A., West, J. L., & Suh, J. (2011) · Soft Matter · doi:10.1039/c0sm01209b · Cited by 10

Gene Therapies for Hepatitis C Virus

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Chimeric AAV Cap sequences alter gene transduction

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A directed evolution approach to select for novel Adeno-associated virus capsids on an HIV-1 producer T cell line

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Preparation and Administration of Adeno-associated Virus Vectors for Corneal Gene Delivery

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Disease Modification Through Trophic Factor Delivery

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Preexisting Neutralizing Antibodies against Different Adeno-Associated Virus Serotypes in Humans and Large Animal Models for Gene Therapy

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Enhanced transgene expression from single-stranded AAV vectors in human cells in vitro and in murine hepatocytes in vivo

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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 9

Identification and validation of novel engineered AAV capsid variants targeting human glia

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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

Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy

Okada, T. (2013) · InTech eBooks · doi:10.5772/53023 · Cited by 7

A Mechanistic Approach to the Development of Gene Therapy for Chronic Pain

Kibaly, C., Loh, H. H., & Law, P. (2016) · International review of cytology · doi:10.1016/bs.ircmb.2016.06.002 · Cited by 7

Adeno-associated virus serotypes 7 and 8 outperform serotype 9 in expressing atheroprotective human apoE3 from mouse skeletal muscle

Evans, V. C., Graham, I. R., Athanasopoulos, T., Galley, D. J., Jackson, C. L., Simons, J. P., Dickson, G., & Owen, J. S. (2010) · Metabolism · doi:10.1016/j.metabol.2010.04.015 · Cited by 6

ShuffleAnalyzer: A Comprehensive Tool to Visualize DNA Shuffling

Schweiggert, F., Habeck, G., Most, P., Busch, M., & Schweiggert, J. (2024) · ACS Synthetic Biology · doi:10.1021/acssynbio.4c00251 · Cited by 6

Gene Therapy “Made in Germany”: A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene Therapy

Büning, H., Fehse, B., Ivics, Z., Kochanek, S., Koehl, U., Kupatt, C., Mussolino, C., Nettelbeck, D. M., Schambach, A., Uckert, W., et al. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.29178.hbu · Cited by 6

Boosters for adeno-associated virus (AAV) vector (r)evolution

Szumska, J., & Grimm, D. (2022) · Cytotherapy · doi:10.1016/j.jcyt.2022.07.005 · Cited by 7

Identification of Novel Retinal Pericyte-Targeting rAAV Vectors Through Directed Evolution

Patel, D. D., Marsic, D., Periasamy, R., Zolotukhin, S., & Lipinski, D. M. (2022) · Translational Vision Science & Technology · doi:10.1167/tvst.11.8.28 · Cited by 6

The identity of the cell adhesive protein substrate affects the efficiency of adeno-associated virus reverse transduction

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Nab Escaping AAV Mutants Isolated from Mouse Muscles

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A Precise Chemical Strategy To Alter the Receptor Specificity of the Adeno‐Associated Virus

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Viral vectors for gene delivery to the central nervous system

Ingusci, S., Hall, B. L., Goins, W. F., Cohen, J. B., & Glorioso, J. C. (2024) · Handbook of clinical neurology · doi:10.1016/b978-0-323-90120-8.00001-0 · Cited by 7

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

Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS

Smith, L. J., Schulman, L., Smith, S. E., van Lieshout, L. P., Barnés, C. M., Behmoiras, L., Scarpitti, M., Kivaa, M., Duong, K. L., Benard, L. O., et al. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.06.013 · Cited by 5

mGem: AAV, from almost a virus to an awesome vector—or is it?

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Adeno-associated Virus as a Mammalian DNA Vector

Salganik, M., Hirsch, M. L., & Samulski, R. J. (2015) · ASM Press eBooks · doi:10.1128/9781555819217.ch37 · Cited by 5

In This Issue

(2008) · Molecular Therapy · doi:10.1038/mt.2008.139

743. Directed Evolution of Adeno-Associated Virus (AAV) by DNA Shuffling Yields Enhanced Gene Delivery Vectors

Li, W., & Samulski, J. R. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.08.825 · Cited by 2

Dissection and optimization of Adeno-associated virus (AAV) DNA family shuffling technology: The journey is the reward

Herrmann, A.-K. (2018) · heiDOK (Heidelberg University) · doi:10.11588/heidok.00025503

Secrets to finding the ideal mate:New insights into parameters that govern successful Adeno-associated virus (AAV) vector evolution

Kienle, E. (2014) · heiDOK (Heidelberg University) · doi:10.11588/heidok.00017851 · Cited by 1

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

Kienle, E., Senís, E., Börner, K., Niopek, D., Wiedtke, E., Große, S., & Grimm, D. (2012) · Journal of Visualized Experiments · doi:10.3791/3819 · Cited by 31