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Empty virions in AAV8 vector preparations reduce transduction efficiency and may cause total viral particle dose-limiting side effects
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What this work says
Outgoing claims: the papers this work cites, builds on, or argues with.
inferredcites →https://doi.org/10.1002/9780471729259.mc14d01s26openalex-snapshot
Production and Discovery of Novel Recombinant Adeno‐Associated Viral Vectors
inferredcites →https://doi.org/10.1002/9780471729259.mc14d06s29openalex-snapshot
Gene Transfer in the Liver Using Recombinant Adeno‐Associated Virus
inferredcites →https://doi.org/10.1002/hep.23043openalex-snapshot
Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver†
inferredcites →https://doi.org/10.1002/jcb.10053openalex-snapshot
Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors
inferredcites →https://doi.org/10.1002/jps.21008openalex-snapshot
Determination of Particle Heterogeneity and Stability of Recombinant Adenovirus by Analytical Ultracentrifugation in CsCl Gradients
inferredcites →https://doi.org/10.1016/j.ymthe.2005.03.020openalex-snapshot
No Evidence for Tumorigenesis of AAV Vectors in a Large-Scale Study in Mice
inferredcites →https://doi.org/10.1016/j.ymthe.2006.03.008openalex-snapshot
Analysis of Tumors Arising in Male B6C3F1 Mice with and without AAV Vector Delivery to Liver
inferredcites →https://doi.org/10.1016/s0091-679x(07)84006-4openalex-snapshot
Analytical Ultracentrifugation: Sedimentation Velocity and Sedimentation Equilibrium
inferredcites →https://doi.org/10.1017/s1431927610055716openalex-snapshot
Transmission Electron Tomography of Adeno-Associated Virus (AAV)
inferredcites →https://doi.org/10.1038/gt.2009.157openalex-snapshot
High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency
inferredcites →https://doi.org/10.1038/mt.2009.301openalex-snapshot
BALB/c Mice Show Impaired Hepatic Tolerogenic Response Following AAV Gene Transfer to the Liver
inferredcites →https://doi.org/10.1038/mt.2011.72openalex-snapshot
Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates
inferredcites →https://doi.org/10.1038/mt.2013.138openalex-snapshot
A Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan Mice
inferredcites →https://doi.org/10.1038/mt.2013.218openalex-snapshot
CD8+ T Cell Recognition of Epitopes Within the Capsid of Adeno-associated Virus 8–based Gene Transfer Vectors Depends on Vectors’ Genome
inferredcites →https://doi.org/10.1038/ng0797-270openalex-snapshot
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
inferredcites →https://doi.org/10.1038/nm1358openalex-snapshot
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
inferredcites →https://doi.org/10.1038/nm1549openalex-snapshot
CD8+ T-cell responses to adeno-associated virus capsid in humans
inferredcites →https://doi.org/10.1056/nejmoa1108046openalex-snapshot
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
inferredcites →https://doi.org/10.1073/pnas.81.20.6466openalex-snapshot
Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.
inferredcites →https://doi.org/10.1073/pnas.90.22.10613openalex-snapshot
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.
inferredcites →https://doi.org/10.1089/hum.2010.202openalex-snapshot
Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial
inferredcites →https://doi.org/10.1089/hum.2013.065openalex-snapshot
Kinetics of Adeno-Associated Virus Serotype 2 (AAV2) and AAV8 Capsid Antigen Presentation In Vivo Are Identical
inferredcites →https://doi.org/10.1101/pdb.top095513openalex-snapshot
Introducing Genes into Mammalian Cells: Viral Vectors
inferredcites →https://doi.org/10.1126/scitranslmed.3005795openalex-snapshot
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
inferredcites →https://doi.org/10.1128/jvi.02524-12openalex-snapshot
Structure and Dynamics of Adeno-Associated Virus Serotype 1 VP1-Unique N-Terminal Domain and Its Role in Capsid Trafficking
inferredcites →https://doi.org/10.1128/jvi.70.11.8098-8108.1996openalex-snapshot
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
inferredcites →https://doi.org/10.1172/jci66611openalex-snapshot
Adeno-associated virus capsid antigen presentation is dependent on endosomal escape
inferredcites →https://doi.org/10.1172/jci68205openalex-snapshot
CpG-depleted adeno-associated virus vectors evade immune detection
inferredcites →https://doi.org/10.1182/blood-2010-08-302729openalex-snapshot
Assessing the potential for AAV vector genotoxicity in a murine model
inferredcites →https://doi.org/10.2174/1566523013348788openalex-snapshot
Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy
inferredcites →https://doi.org/10.2174/1566523054065057openalex-snapshot
New Recombinant Serotypes of AAV Vectors
What points at this work
Incoming claims: where this work appears in other papers’ reference lists and arguments.
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Separation of Full and Empty AAV Capsids by Anion-Exchange Chromatography Using Choline-Type Salts
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Biomarker and Bioanalytical Readouts for the Development of AAV Gene Therapy
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Quantitative Assessment of the Physical Virus Titer and Purity by Ultrasensitive Flow Virometry
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Quantitative Assessment of the Physical Virus Titer and Purity by Ultrasensitive Flow Virometry
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Approaches to purification and concentration of rAAV vectors for gene therapy
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Separating Empty and Full Recombinant Adeno‐Associated Virus Particles Using Isocratic Anion Exchange Chromatography
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Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors
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Novel anion exchange membrane chromatography method for the separation of empty and full adeno‐associated virus
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Tuning mobile phase properties to improve empty and full particle separation in adeno‐associated virus productions by anion exchange chromatography
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Tuning capsid formation dynamics in recombinant adeno‐associated virus producing synthetic cell lines to enhance full particle productivity
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A Novel and Simplified Anion Exchange Flow‐Through Polishing Approach for the Separation of Full From Empty Adeno‐Associated Virus Capsids
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Tracking Particle‐Encapsulated DNA Across the Anion‐Exchange Chromatography Fractions of Recombinant Adeno‐Associated Virus Using Droplet Digital PCR and High‐Throughput Sequencing
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Anion‐Exchange Membrane Chromatography for the Separation of Empty and Full Adeno‐Associated Viral Capsids
inferred← citeshttps://doi.org/10.1002/bit.28412openalex-snapshot
Critical challenges and advances in recombinant adeno‐associated virus (rAAV) biomanufacturing
inferred← citeshttps://doi.org/10.1002/bit.28453openalex-snapshot
Enrichment of adeno‐associated virus serotype 5 full capsids by anion exchange chromatography with dual salt elution gradients
inferred← citeshttps://doi.org/10.1002/bit.28828openalex-snapshot
Multidose transient transfection of human embryonic kidney 293 cells modulates recombinant adeno‐associated virus2/5 Rep protein expression and influences the enrichment fraction of filled capsids
inferred← citeshttps://doi.org/10.1002/bit.28885openalex-snapshot
Expression of Viral DNA Polymerase in Synthetic Recombinant Adeno‐Associated Virus Producer Cell Line Enhances Full Particle Productivity
inferred← citeshttps://doi.org/10.1002/bit.28932openalex-snapshot
Chromatography in Downstream Processing of Recombinant Adeno‐Associated Viruses: A Review of Current and Future Practises
inferred← citeshttps://doi.org/10.1002/bit.28980openalex-snapshot
Development of an HEK293 Suspension Cell Culture Medium, Transient Transfection Optimization Workflow, and Analytics for Batch rAAV Manufacturing
inferred← citeshttps://doi.org/10.1002/bit.70021openalex-snapshot
Manufacturing Process Intensification of Adeno‐Associated Viral Vectors Type‐8 Using Weak Partitioning Chromatography With UV‐Based Process Analytical Technology (PAT)
inferred← citeshttps://doi.org/10.1002/btpr.70065openalex-snapshot
Enrichment of full AAV capsids by preparative strong anion exchange chromatography
inferred← citeshttps://doi.org/10.1002/smll.202409353openalex-snapshot
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
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CMC Considerations for Continuous Bioprocess Design, Development, and Manufacturing
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A novel scalable, robust downstream process for oncolytic rat parvovirus: isoelectric point-based elimination of empty particles
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Production of retroviral vectors in continuous high cell density culture
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Hybrid modeling of an ultracentrifugation process for separation of full and empty adeno-associated virus particles
inferred← citeshttps://doi.org/10.1007/s10928-023-09889-1openalex-snapshot
Prospective approaches to gene therapy computational modeling – spotlight on viral gene therapy
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Gene Therapy for Hemophilia: Progress to Date
inferred← citeshttps://doi.org/10.1007/s40259-023-00585-7openalex-snapshot
Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer
inferred← citeshttps://doi.org/10.1016/b978-0-443-18748-3.00012-3openalex-snapshot
Product process initiations of biopharmaceuticals
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Multi-Wavelength Analytical Ultracentrifugation of Biopolymer Mixtures and Interactions
inferred← citeshttps://doi.org/10.1016/j.ab.2023.115421openalex-snapshot
Separation of full and empty adeno-associated virus capsids by anion-exchange chromatography using choline-type salts
inferred← citeshttps://doi.org/10.1016/j.bbrep.2025.102397openalex-snapshot
Methodological validation of a cryo-TEM-based detection method for empty capsid ratio in recombinant adeno-associated virus
inferred← citeshttps://doi.org/10.1016/j.biotechadv.2021.107764openalex-snapshot
Cellular pathways of recombinant adeno-associated virus production for gene therapy
inferred← citeshttps://doi.org/10.1016/j.chroma.2021.462210openalex-snapshot
Removal of empty capsids from adeno-associated virus preparations by multimodal metal affinity chromatography
inferred← citeshttps://doi.org/10.1016/j.chroma.2025.465674openalex-snapshot
Combining steric exclusion with anion exchange – development of a universal and scalable adeno-associated virus downstream process
inferred← citeshttps://doi.org/10.1016/j.chroma.2025.466219openalex-snapshot
Separation of empty and full adeno-associated viral capsids by MCSGP: From model-based design to implementation
inferred← citeshttps://doi.org/10.1016/j.chroma.2025.466644openalex-snapshot
Selectivity for full AAV capsids in affinity capture with camelid ligands
inferred← citeshttps://doi.org/10.1016/j.chroma.2026.466838openalex-snapshot
Advances in chromatography for biomacromolecules and bioparticles (2019–2024): Focus on materials and industrial applications
inferred← citeshttps://doi.org/10.1016/j.cobme.2021.100353openalex-snapshot
AAV manufacturing for clinical use: Insights on current challenges from the upstream process perspective
inferred← citeshttps://doi.org/10.1016/j.drudis.2022.103442openalex-snapshot
Mass spectrometry in gene therapy: Challenges and opportunities for AAV analysis
inferred← citeshttps://doi.org/10.1016/j.ejpb.2020.08.002openalex-snapshot
Optimization of the quality by design approach for gene therapy products: A case study for adeno-associated viral vectors
inferred← citeshttps://doi.org/10.1016/j.ejpb.2023.05.011openalex-snapshot
Purity and DNA content of AAV capsids assessed by analytical ultracentrifugation and orthogonal biophysical techniques
inferred← citeshttps://doi.org/10.1016/j.heliyon.2023.e15071openalex-snapshot
AAV genome modification for efficient AAV production
inferred← citeshttps://doi.org/10.1016/j.jchromb.2024.124206openalex-snapshot
Separation of full, empty, and partial adeno-associated virus capsids via anion-exchange chromatography with continuous recycling and accumulation
inferred← citeshttps://doi.org/10.1016/j.jpba.2026.117524openalex-snapshot
Optimizing LOD/LOQ and sample consumption during analytical ultracentrifugation to characterize AAV gene therapy vectors
inferred← citeshttps://doi.org/10.1016/j.jve.2023.100316openalex-snapshot
Adeno-associated virus-vectored delivery of HIV biologics: the promise of a “single-shot” functional cure for HIV infection
inferred← citeshttps://doi.org/10.1016/j.jviromet.2015.11.022openalex-snapshot
Standardized large-scale H-1PV production process with efficient quality and quantity monitoring
inferred← citeshttps://doi.org/10.1016/j.omta.2025.201656openalex-snapshot
Role of viral protein ratio in the structure and separation of empty and full adeno-associated virus capsids: A molecular dynamics study
inferred← citeshttps://doi.org/10.1016/j.omta.2026.201700openalex-snapshot
Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors
inferred← citeshttps://doi.org/10.1016/j.omta.2026.201770openalex-snapshot
Intracellular trafficking and nuclear compartmentalization of AAV gene delivery
inferred← citeshttps://doi.org/10.1016/j.omta.2026.201787openalex-snapshot
AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality
inferred← citeshttps://doi.org/10.1016/j.omtm.2016.12.004openalex-snapshot
Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
inferred← citeshttps://doi.org/10.1016/j.omtm.2019.09.006openalex-snapshot
Developing an Anion Exchange Chromatography Assay for Determining Empty and Full Capsid Contents in AAV6.2
inferred← citeshttps://doi.org/10.1016/j.omtm.2020.06.003openalex-snapshot
Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity
inferred← citeshttps://doi.org/10.1016/j.omtm.2021.04.003openalex-snapshot
Anion-exchange HPLC assay for separation and quantification of empty and full capsids in multiple adeno-associated virus serotypes
inferred← citeshttps://doi.org/10.1016/j.omtm.2021.08.002openalex-snapshot
Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry
inferred← citeshttps://doi.org/10.1016/j.omtm.2023.07.007openalex-snapshot
Partial genome content within rAAVs impacts performance in a cell assay-dependent manner
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Adeno-associated viral capsid stability on anion exchange chromatography column and its impact on empty and full capsid separation
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A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies
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Fast HPLC-based affinity method to determine capsid titer and full/empty ratio of adeno-associated viral vectors
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Cell-penetrating peptide-grafted AAV2 capsids for improved retinal delivery via intravitreal injection
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A two-pass anion-exchange chromatography strategy for enrichment of full capsids in manufacturing of adeno-associated viral vectors
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AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101581openalex-snapshot
Variation of VP2 stoichiometry and deamidation of VP1 during production and their impacts on the transduction efficiency of AAV vectors
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101607openalex-snapshot
End-to-end characterization of AAV manufacturing process using charge detection mass spectrometry
inferred← citeshttps://doi.org/10.1016/j.slast.2026.100404openalex-snapshot
Viral vector research in human gene therapy: Basic principles, alternative evaluation models, and clinical applications
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Chromatographic strategies for the analytical characterization of adeno-associated virus vector-based gene therapy products
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Comprehensive Size Distribution and Composition Analysis of Adeno-Associated Virus Vector by Multiwavelength Sedimentation Velocity Analytical Ultracentrifugation
inferred← citeshttps://doi.org/10.1016/j.xphs.2022.10.023openalex-snapshot
Size Distribution Analysis of the Adeno-Associated Virus Vector by the c(s) Analysis of Band Sedimentation Analytical Ultracentrifugation with Multiwavelength Detection
inferred← citeshttps://doi.org/10.1016/j.xphs.2024.03.026openalex-snapshot
Combined 100 keV Cryo-Electron Microscopy and Image Analysis Methods to Characterize the Wider Adeno-Associated Viral Products
inferred← citeshttps://doi.org/10.1016/j.xphs.2025.103878openalex-snapshot
Particle size distribution resolution limit of AAV high-speed sedimentation velocity protocol relative to low-speed sedimentation velocity protocol, mass photometry, and charge-detection mass spectrometry
inferred← citeshttps://doi.org/10.1016/j.xphs.2025.103899openalex-snapshot
Impact of electrostatics on the aggregation, genome release, and self-interactions of AAV9 capsids
inferred← citeshttps://doi.org/10.1016/j.ymthe.2023.10.005openalex-snapshot
Redirecting AAV vectors to extrahepatic tissues
inferred← citeshttps://doi.org/10.1016/j.ymthe.2024.03.036openalex-snapshot
An innate immune response to adeno-associated virus genomes decreases cortical dendritic complexity and disrupts synaptic transmission
inferred← citeshttps://doi.org/10.1016/j.ymthe.2025.03.037openalex-snapshot
The curious case of AAV immunology
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A Native Mass Spectrometry-Based Assay for Rapid Assessment of the Empty:Full Capsid Ratio in Adeno-Associated Virus Gene Therapy Products
inferred← citeshttps://doi.org/10.1021/acs.analchem.1c04088openalex-snapshot
Rapid and Noninvasive Quantification of Capsid Gene Filling Level Using Water Proton Nuclear Magnetic Resonance
inferred← citeshttps://doi.org/10.1021/acs.analchem.1c04873openalex-snapshot
Development of a Two-Dimensional Liquid Chromatography-Mass Spectrometry Platform for Simultaneous Multi-Attribute Characterization of Adeno-Associated Viruses
inferred← citeshttps://doi.org/10.1021/acs.analchem.3c01955openalex-snapshot
Applications and Limitations of Equilibrium Density Gradient Analytical Ultracentrifugation for the Quantitative Characterization of Adeno-Associated Virus Vectors
inferred← citeshttps://doi.org/10.1021/acs.analchem.4c05229openalex-snapshot
Multimass Analysis of Adeno-Associated Virus Vectors by Orbitrap-Based Charge Detection Mass Spectrometry
inferred← citeshttps://doi.org/10.1021/acs.analchem.4c06397openalex-snapshot
A Robust and Automated Platform for Charge Detection Mass Spectrometry of Megadalton Biotherapeutics
inferred← citeshttps://doi.org/10.1021/acs.analchem.6b00883openalex-snapshot
Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry
inferred← citeshttps://doi.org/10.1021/acs.langmuir.3c00241openalex-snapshot
Distinguishing between DNA-Loaded Full and Empty Capsids of Adeno-Associated Virus with Atomic Force Microscopy Imaging
inferred← citeshttps://doi.org/10.1021/acsnano.5c00566openalex-snapshot
Selective Enrichment of Full Capsids of Adeno-Associated Virus
inferred← citeshttps://doi.org/10.1021/jacs.4c13393openalex-snapshot
Prolonged Trapping of Adeno-Associated Virus Capsids Reveals that Genome Packaging Affects Single-Ion Mass Spectrometry Measurements
inferred← citeshttps://doi.org/10.1038/mt.2013.268openalex-snapshot
AAV Empty Capsids: For Better or for Worse?
inferred← citeshttps://doi.org/10.1038/mt.2015.134openalex-snapshot
Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer
inferred← citeshttps://doi.org/10.1038/mtm.2016.19openalex-snapshot
Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR
inferred← citeshttps://doi.org/10.1038/mtna.2015.32openalex-snapshot
Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing
inferred← citeshttps://doi.org/10.1038/mtna.2016.49openalex-snapshot
CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector
inferred← citeshttps://doi.org/10.1038/s41434-018-0034-7openalex-snapshot
Optimization of viral protein ratios for production of rAAV serotype 5 in the baculovirus system
inferred← citeshttps://doi.org/10.1038/s41434-019-0104-5openalex-snapshot
Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
inferred← citeshttps://doi.org/10.1038/s41434-021-00299-xopenalex-snapshot
A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging
inferred← citeshttps://doi.org/10.1038/s41434-021-00311-4openalex-snapshot
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry
inferred← citeshttps://doi.org/10.1038/s41434-024-00444-2openalex-snapshot
Analytical characterization of full, intermediate, and empty AAV capsids
inferred← citeshttps://doi.org/10.1038/s41434-025-00546-5openalex-snapshot
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production
inferred← citeshttps://doi.org/10.1038/s41467-017-00406-wopenalex-snapshot
Regulation of RIPK1 activation by TAK1-mediated phosphorylation dictates apoptosis and necroptosis
inferred← citeshttps://doi.org/10.1038/s41596-018-0097-3openalex-snapshot
Systemic AAV vectors for widespread and targeted gene delivery in rodents
inferred← citeshttps://doi.org/10.1038/s41598-019-46069-zopenalex-snapshot
The adeno-associated virus rh10 vector is an effective gene transfer system for chronic spinal cord injury
inferred← citeshttps://doi.org/10.1038/s44321-025-00248-wopenalex-snapshot
High-purity AAV vector production utilizing recombination-dependent minicircle formation and genetic coupling
inferred← citeshttps://doi.org/10.1039/d3ay01560bopenalex-snapshot
Mass spectrometry friendly pH-gradient anion exchange chromatography for the separation of full and empty adeno-associated virus (AAV) capsids
inferred← citeshttps://doi.org/10.1042/etls20210179openalex-snapshot
Manufacturing of AAV vectors: translational challenges from development to industrialisation
inferred← citeshttps://doi.org/10.1053/j.seminhematol.2015.10.002openalex-snapshot
Gene therapy for hemophilia: past, present and future
inferred← citeshttps://doi.org/10.1089/hgtb.2015.048openalex-snapshot
Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors
inferred← citeshttps://doi.org/10.1089/hgtb.2016.188openalex-snapshot
Improving the Quality of Adeno-Associated Viral Vector Preparations: The Challenge of Product-Related Impurities
inferred← citeshttps://doi.org/10.1089/hgtb.2019.105openalex-snapshot
Lot-to-Lot Variation in Adeno-Associated Virus Serotype 9 (AAV9) Preparations
inferred← citeshttps://doi.org/10.1089/hum.2018.018openalex-snapshot
CRISPR/Cas9 Inhibits Multiple Steps of HIV-1 Infection
inferred← citeshttps://doi.org/10.1089/hum.2019.041openalex-snapshot
Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy
inferred← citeshttps://doi.org/10.1089/hum.2019.144openalex-snapshot
Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and Capsid
inferred← citeshttps://doi.org/10.1089/hum.2021.123openalex-snapshot
Size Exclusion Chromatography with Dual Wavelength Detection as a Sensitive and Accurate Method for Determining the Empty and Full Capsids of Recombinant Adeno-Associated Viral Vectors
inferred← citeshttps://doi.org/10.1089/hum.2022.116openalex-snapshot
Significant Differences in Capsid Properties and Potency Between Adeno-Associated Virus Vectors Produced in Sf9 and HEK293 Cells
inferred← citeshttps://doi.org/10.1089/hum.2022.163openalex-snapshot
Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration
inferred← citeshttps://doi.org/10.1089/hum.2022.218openalex-snapshot
Application of Size Exclusion Chromatography with Multiangle Light Scattering in the Analytical Development of a Preclinical Stage Gene Therapy Program
inferred← citeshttps://doi.org/10.1089/hum.2023.169openalex-snapshot
Methodological Validation of Sedimentation Velocity Analytical Ultracentrifugation Method for Adeno-Associated Virus and Collaborative Calibration of System Suitability Substance
inferred← citeshttps://doi.org/10.1089/hum.2024.115openalex-snapshot
Evaluation of Purification Methods for Minimizing Transgene Expression Background During Viral Manufacturing
inferred← citeshttps://doi.org/10.1089/hum.2024.124openalex-snapshot
Interlaboratory Measurement of Adeno-Associated Virus: Comparative Quantification of Full and Empty Capsids
inferred← citeshttps://doi.org/10.1089/hum.2024.162openalex-snapshot
A Single-Sector Higher Throughput Sedimentation Velocity Analytical Ultracentrifugation Method for Recombinant Adeno-Associated Virus Empty and Full Ratio Analysis
inferred← citeshttps://doi.org/10.1093/ejhf/xuag105openalex-snapshot
AB-1002 gene therapy expressing active protein phosphatase inhibitor-1 in heart failure: rationale and study design of the GenePHIT trial
inferred← citeshttps://doi.org/10.1093/jac/dkw104openalex-snapshot
Pharmacodynamics of anti-HIV gene therapy using viral vectors and targeted endonucleases
inferred← citeshttps://doi.org/10.1101/2021.07.06.451264openalex-snapshot
A native mass spectrometry-based assay for rapid assessment of the empty/full capsid ratio in AAV gene therapy products
inferred← citeshttps://doi.org/10.1101/2022.08.27.505551openalex-snapshot
Spectral Interferences Impede the High-Resolution Mass Analysis of Recombinant Adeno-Associated Viruses
inferred← citeshttps://doi.org/10.1101/246405openalex-snapshot
Widespread and targeted gene expression by systemic AAV vectors: Production, purification, and administration
inferred← citeshttps://doi.org/10.1101/cshperspect.a041307openalex-snapshot
Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl
inferred← citeshttps://doi.org/10.1111/brv.12718openalex-snapshot
Humoral immune responses to AAV gene therapy in the ocular compartment
inferred← citeshttps://doi.org/10.1128/jvi.00773-21openalex-snapshot
Improved Genome Packaging Efficiency of Adeno-associated Virus Vectors Using Rep Hybrids
inferred← citeshttps://doi.org/10.1128/spectrum.01806-24openalex-snapshot
Identification of relevant analytical methods for adeno-associated virus stability assessment during formulation development
inferred← citeshttps://doi.org/10.1177/01926233211041962openalex-snapshot
Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors
inferred← citeshttps://doi.org/10.1177/10430342251378524openalex-snapshot
Pre-Existing Anti-Adeno-Associated Virus Immunity in Gene Therapy: Mechanisms, Challenges, and Potential Solutions
inferred← citeshttps://doi.org/10.1177/10430342251408498openalex-snapshot
Exploring Development Options of a Polishing Chromatography Step for AAV7 and AAV8
inferred← citeshttps://doi.org/10.1208/s12248-025-01194-8openalex-snapshot
Quantifying the Full-to-Empty Adeno-Associated Virus (AAV) Capsid Ratios and Their Impact on Transduction Efficiency in vitro
inferred← citeshttps://doi.org/10.1208/s12249-025-03220-6openalex-snapshot
Integrated Analytical Techniques to Investigate the Effect of the Freezing/Thawing Cycles on the Non-replicating Recombinant Chimpanzee Adenovirus Viral Vector COVID-19 Vaccine
inferred← citeshttps://doi.org/10.1371/journal.pone.0194728openalex-snapshot
Ultrafiltered recombinant AAV8 vector can be safely administered in vivo and efficiently transduces liver
inferred← citeshttps://doi.org/10.1371/journal.pone.0269139openalex-snapshot
Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM
inferred← citeshttps://doi.org/10.1517/14712598.2015.1035645openalex-snapshot
Strategies to circumvent humoral immunity to adeno-associated viral vectors
inferred← citeshttps://doi.org/10.1517/21678707.2015.1069179openalex-snapshot
Obstacles and future of gene therapy for hemophilia
inferred← citeshttps://doi.org/10.17615/cr64-be62openalex-snapshot
Biology of AAV Vectors in the Central Nervous System
inferred← citeshttps://doi.org/10.21203/rs.3.rs-1184379/v1openalex-snapshot
Multi-Wavelength Analytical Ultracentrifugation of Biopolymer Mixtures and Interactions
inferred← citeshttps://doi.org/10.30895/2221-996x-2025-580openalex-snapshot
Implementation of the quality-by-design concept for an adeno-associated viral vector-based gene therapy
inferred← citeshttps://doi.org/10.3389/fbioe.2024.1436857openalex-snapshot
An improved high-resolution method for quantitative separation of empty and filled AAV8 capsids by strong anion exchange HPLC
inferred← citeshttps://doi.org/10.3389/fimmu.2014.00350openalex-snapshot
Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions
inferred← citeshttps://doi.org/10.3389/fimmu.2018.00844openalex-snapshot
Efficient Capsid Antigen Presentation From Adeno-Associated Virus Empty Virions In Vivo
inferred← citeshttps://doi.org/10.3389/fimmu.2022.991832openalex-snapshot
A versatile toolkit for overcoming AAV immunity
inferred← citeshttps://doi.org/10.3389/fphar.2022.815317openalex-snapshot
Chimeric Mice Engrafted With Canine Hepatocytes Exhibits Similar AAV Transduction Efficiency to Hemophilia B Dog
inferred← citeshttps://doi.org/10.3390/biomedicines12071523openalex-snapshot
Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects
inferred← citeshttps://doi.org/10.3390/biomedicines12092128openalex-snapshot
Structural Changes Likely Cause Chemical Differences between Empty and Full AAV Capsids
inferred← citeshttps://doi.org/10.3390/biomedicines13020361openalex-snapshot
Consecutive Affinity and Ion-Exchange Chromatography for AAV9 Vectors Purification
inferred← citeshttps://doi.org/10.3390/cells12182264openalex-snapshot
Size Exclusion Chromatography–Mass Photometry: A New Method for Adeno-Associated Virus Product Characterization
inferred← citeshttps://doi.org/10.3390/ijms232012332openalex-snapshot
Anion-Exchange Chromatography at the Service of Gene Therapy: Baseline Separation of Full/Empty Adeno-Associated Virus Capsids by Screening of Conditions and Step Gradient Elution Mode
inferred← citeshttps://doi.org/10.3390/ijms25052785openalex-snapshot
A Direct Comparison of rAAV5 Variants Derived from the Baculovirus Expression System Using LC-MS Workflows Demonstrates Key Differences in Overall Production Yield, Product Quality and Vector Efficiency
inferred← citeshttps://doi.org/10.3390/ijms25158342openalex-snapshot
Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation
inferred← citeshttps://doi.org/10.3390/ijms26104941openalex-snapshot
Genomic Size Is Critical to Guarantee the Genomic Stability of Non-Replicative HSV1 Vectors
inferred← citeshttps://doi.org/10.3390/ijms27114818openalex-snapshot
Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization
inferred← citeshttps://doi.org/10.3390/jpm10040258openalex-snapshot
Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back
inferred← citeshttps://doi.org/10.3390/membranes14090194openalex-snapshot
A Novel Method for Separating Full and Empty Adeno-Associated Viral Capsids Using Ultrafiltration
inferred← citeshttps://doi.org/10.3390/molecules24213973openalex-snapshot
A Generic Method for Fast and Sensitive Detection of Adeno-Associated Viruses Using Modified AAV Receptor Recombinant Proteins
inferred← citeshttps://doi.org/10.3390/standards6040039openalex-snapshot
Reference-Anchored Equivalence: A Unified Scientific Framework for the Approval of Follow-On Drugs and Biological Products
inferred← citeshttps://doi.org/10.3390/v13071336openalex-snapshot
Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
inferred← citeshttps://doi.org/10.3390/v14081623openalex-snapshot
Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method
inferred← citeshttps://doi.org/10.3390/v16010051openalex-snapshot
A Lac Repressor-Inducible Baculovirus Expression Vector for Controlling Adeno-Associated Virus Capsid Ratios
inferred← citeshttps://doi.org/10.3390/v17020239openalex-snapshot
Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy
inferred← citeshttps://doi.org/10.53846/goediss-8451openalex-snapshot
Molecular mechanisms of TBX5-related conduction disorders
inferred← citeshttps://doi.org/10.64898/2025.12.09.693273openalex-snapshot
Insights into the AAV packaging mechanism: Cryo-EM Structure of the AAV2 Rep-Capsid Packaging Complex
inferred← citeshttps://doi.org/10.64898/2026.05.21.726733openalex-snapshot
YY1 Binding Motif Upstream of Rep/Cap Increases AAV Yield and Full Capsids
inferred← citeshttps://doi.org/10.70675/c5cff6daz8fe4z430cz80e6z97a11cc59c8eopenalex-snapshot
Characterization of rAAV vectors packaging in baculovirusinfected insect cells
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