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Quality Control Testing, Characterization and Critical Quality Attributes of Adeno‐Associated Virus Vectors Used for Human Gene Therapy

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What this work says

Outgoing claims: the papers this work cites, builds on, or argues with.

inferredcites →https://doi.org/10.1007/978-1-61779-095-9_11openalex-snapshot
Vector Characterization Methods for Quality Control Testing of Recombinant Adeno-Associated Viruses
inferredcites →https://doi.org/10.1016/j.immuni.2018.03.013openalex-snapshot
Toll-like Receptor 9 Contains Two DNA Binding Sites that Function Cooperatively to Promote Receptor Dimerization and Activation
inferredcites →https://doi.org/10.1016/j.omtm.2018.01.002openalex-snapshot
Pharmacology of Recombinant Adeno-associated Virus Production
inferredcites →https://doi.org/10.1016/j.ymthe.2005.06.003openalex-snapshot
Evidence for Encapsidation of Prokaryotic Sequences during Recombinant Adeno-Associated Virus Production and Their in Vivo Persistence after Vector Delivery
inferredcites →https://doi.org/10.1016/j.ymthe.2018.09.013openalex-snapshot
Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function
inferredcites →https://doi.org/10.1016/j.ymthe.2019.11.011openalex-snapshot
Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells
inferredcites →https://doi.org/10.1016/j.ymthe.2019.11.014openalex-snapshot
The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer
inferredcites →https://doi.org/10.1016/j.ymthe.2020.01.026openalex-snapshot
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?
inferredcites →https://doi.org/10.1016/j.ymthe.2020.07.009openalex-snapshot
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV
inferredcites →https://doi.org/10.1038/mt.2008.227openalex-snapshot
Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses
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/10.1038/nrg2988openalex-snapshot
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
inferredcites →https://doi.org/10.1038/s41576-019-0205-4openalex-snapshot
Engineering adeno-associated virus vectors for gene therapy
inferredcites →https://doi.org/10.1073/pnas.162250899openalex-snapshot
The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy
inferredcites →https://doi.org/10.1073/pnas.96.16.9305openalex-snapshot
CpG DNA: A potent signal for growth, activation, and maturation of human dendritic cells
inferredcites →https://doi.org/10.1089/hum.2011.160openalex-snapshot
Native Molecular State of Adeno-Associated Viral Vectors Revealed by Single-Molecule Sequencing
inferredcites →https://doi.org/10.1089/hum.2020.182openalex-snapshot
Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
inferredcites →https://doi.org/10.1099/0022-1317-83-5-973openalex-snapshot
The VP1 capsid protein of adeno-associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity
inferredcites →https://doi.org/10.1126/science.1142658openalex-snapshot
AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma
inferredcites →https://doi.org/10.1172/jci37607openalex-snapshot
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
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/10.3390/biomedicines2010080openalex-snapshot
Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment
inferredcites →https://doi.org/10.3390/v11010038openalex-snapshot
Methylation Status of the Adeno-Associated Virus Type 2 (AAV2)
inferredcites →https://doi.org/10.3390/v11020102openalex-snapshot
Adeno-Associated Virus (AAV) Versus Immune Response

What points at this work

Incoming claims: where this work appears in other papers’ reference lists and arguments.

inferred← citeshttps://doi.org/10.2139/ssrn.4658165openalex-extracted
Adeno-Associated Virus Analysis by Size Exclusion Chromatography within 3 Minutes Using Short Bio-Inert Columns Made with 3 Μm Particles Operated at High Flowrates
inferred← citeshttps://doi.org/10.1002/9781394271566.ch9openalex-snapshot
Viral Vectors in Veterinary Vaccine Development
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Preserving Nature's Remedies
inferred← citeshttps://doi.org/10.1002/biot.202000614openalex-snapshot
Biomanufacturing of Gene Therapy Vectors
inferred← citeshttps://doi.org/10.1002/biot.202400051openalex-snapshot
Tuning capsid formation dynamics in recombinant adeno‐associated virus producing synthetic cell lines to enhance full particle productivity
inferred← citeshttps://doi.org/10.1002/biot.202400656openalex-snapshot
Rapid At‐Line AAVX Affinity HPLC: Enabling Process Analytical Technology for Bioprocess Development of Adeno‐Associated Virus Vectors
inferred← citeshttps://doi.org/10.1002/biot.70020openalex-snapshot
Intensification of rAAV Production Based on HEK293 Cell Transient Transfection
inferred← citeshttps://doi.org/10.1002/bit.27905openalex-snapshot
Assessment of quality attributes for adeno‐associated viral vectors
inferred← citeshttps://doi.org/10.1002/bit.28402openalex-snapshot
Process economics evaluation and optimization of adeno‐associated virus downstream processing
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.28508openalex-snapshot
Design space determination to optimize DNA complexation and full capsid formation in transient rAAV manufacturing
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/btpr.88542openalex-snapshot
High‐throughput development and scale‐up of recombinant adeno‐associated virus 5 anion‐exchange chromatography for high‐purity capsid separation
inferred← citeshttps://doi.org/10.1002/elps.202100302openalex-snapshot
Stronger together: Analytical techniques for recombinant adeno associated virus
inferred← citeshttps://doi.org/10.1002/elps.202300127openalex-snapshot
Electrophoretic characterization of LNP/AAV‐encapsulated nucleic acids: Strengths and weaknesses
inferred← citeshttps://doi.org/10.1002/med.22036openalex-snapshot
The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution
inferred← citeshttps://doi.org/10.1007/978-3-031-28489-2_1openalex-snapshot
Bioprocessing and Analytical Development for Virus-Based Therapeutics
inferred← citeshttps://doi.org/10.1007/978-3-031-28489-2_12openalex-snapshot
Product-Related Impurities in Therapeutic Virus Bioprocessing
inferred← citeshttps://doi.org/10.1007/978-3-031-81787-8_5openalex-snapshot
Hope Unlocked: Gene Therapy with Viral Vectors and Gene Editing
inferred← citeshttps://doi.org/10.1007/978-981-96-6306-4_11openalex-snapshot
Validation in Sterile Ophthalmic Formulation
inferred← citeshttps://doi.org/10.1007/s00216-024-05623-zopenalex-snapshot
Development of an optimized SEC method for characterization of genome DNA leakage from adeno-associated virus products
inferred← citeshttps://doi.org/10.1007/s00216-026-06429-xopenalex-snapshot
Classification and quantification of full vs. empty adeno-associated virus reference capsids via infrared attenuated total reflection spectroscopy
inferred← citeshttps://doi.org/10.1016/j.ab.2023.115311openalex-snapshot
Multi-attribute analysis of adeno-associated virus by size exclusion chromatography with fluorescence and triple-wavelength UV detection
inferred← citeshttps://doi.org/10.1016/j.bbadva.2025.100151openalex-snapshot
Quality assessment strategy development and analytical method selection of GMP grade biological drugs for gene and cell therapy
inferred← citeshttps://doi.org/10.1016/j.biotechadv.2024.108391openalex-snapshot
Advances and opportunities in process analytical technologies for viral vector manufacturing
inferred← citeshttps://doi.org/10.1016/j.chroma.2024.464684openalex-snapshot
Adeno-associated virus analysis by size exclusion chromatography within 3 minutes using short bio-inert columns made with 3 µm particles operated at high flowrates
inferred← citeshttps://doi.org/10.1016/j.cobme.2021.100339openalex-snapshot
Overview of analytics needed to support a robust gene therapy manufacturing process
inferred← citeshttps://doi.org/10.1016/j.cobme.2023.100517openalex-snapshot
AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities
inferred← citeshttps://doi.org/10.1016/j.coche.2021.100722openalex-snapshot
Bioprocess monitoring and control: challenges in cell and gene therapy
inferred← citeshttps://doi.org/10.1016/j.cocis.2021.101460openalex-snapshot
Stem cells and their extracellular vesicles as natural and bioinspired carriers for the treatment of neurological disorders
inferred← citeshttps://doi.org/10.1016/j.copbio.2021.06.025openalex-snapshot
Analytical methods to characterize recombinant adeno-associated virus vectors and the benefit of standardization and reference materials
inferred← citeshttps://doi.org/10.1016/j.copbio.2021.06.026openalex-snapshot
Analytics of host cell proteins (HCPs): lessons from biopharmaceutical mAb analysis for Gene therapy products
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.ijpharm.2021.120912openalex-snapshot
Development of a stable lyophilized adeno-associated virus gene therapy formulation
inferred← citeshttps://doi.org/10.1016/j.jbiotec.2024.07.020openalex-snapshot
Orthogonal characterization of rAAV9 reveals unexpected transgene heterogeneity
inferred← citeshttps://doi.org/10.1016/j.jbiotec.2026.03.004openalex-snapshot
MS-based workflow for PTM profiling of rAAV capsid proteins to support bioprocess monitoring and development
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.2023.115397openalex-snapshot
Development and application of potency assays based on genetically modified cells for biological products
inferred← citeshttps://doi.org/10.1016/j.jpba.2023.115581openalex-snapshot
Recent applications of quantitative mass spectrometry in biopharmaceutical process development and manufacturing
inferred← citeshttps://doi.org/10.1016/j.omtm.2022.07.018openalex-snapshot
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper
inferred← citeshttps://doi.org/10.1016/j.omtm.2023.06.004openalex-snapshot
Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate
inferred← citeshttps://doi.org/10.1016/j.omtm.2024.101306openalex-snapshot
Biolayer interferometry for adeno-associated virus capsid titer measurement and applications to upstream and downstream process development
inferred← citeshttps://doi.org/10.1016/j.omtm.2024.101309openalex-snapshot
Characterization of AAV vectors: A review of analytical techniques and critical quality attributes
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101530openalex-snapshot
Orthogonal approaches to AAV vector characterization: Validating quantitative TEM for partially filled particles
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101562openalex-snapshot
Development of LC-MS methods for AAV capsid protein quantification and host cell protein profiling
inferred← citeshttps://doi.org/10.1016/j.omtn.2026.103000openalex-snapshot
Mammalian therapeutic riboswitches: Engineering and custom ligands
inferred← citeshttps://doi.org/10.1016/j.phrs.2026.108104openalex-snapshot
Gene modification: Exploring the potential in treating kidney diseases
inferred← citeshttps://doi.org/10.1016/j.pmatsci.2022.100961openalex-snapshot
Essential cues of engineered polymeric materials regulating gene transfer pathways
inferred← citeshttps://doi.org/10.1016/j.scib.2023.12.006openalex-snapshot
Engineered stem cells by emerging biomedical stratagems
inferred← citeshttps://doi.org/10.1016/j.tibtech.2023.04.002openalex-snapshot
Challenges in scaling up AAV-based gene therapy manufacturing
inferred← citeshttps://doi.org/10.1016/j.trac.2023.117088openalex-snapshot
Chromatographic strategies for the analytical characterization of adeno-associated virus vector-based gene therapy products
inferred← citeshttps://doi.org/10.1016/j.virol.2022.06.013openalex-snapshot
Binding of Coxsackievirus A21 procapsids to immobilized glutathione depends on cell culture conditions during infection
inferred← citeshttps://doi.org/10.1016/j.xphs.2021.06.010openalex-snapshot
Quantitation of Trace Levels of DNA Released from Disrupted Adeno-Associated Virus Gene Therapy Vectors
inferred← citeshttps://doi.org/10.1016/j.xphs.2023.05.009openalex-snapshot
Characterization of Virus Particles and Submicron-Sized Particulate Impurities in Recombinant Adeno-Associated Virus Drug Product
inferred← citeshttps://doi.org/10.1016/j.xphs.2024.03.024openalex-snapshot
Drug product Formulation and Fill/Finish Manufacturing Process Considerations for AAV-Based Genomic Medicines
inferred← citeshttps://doi.org/10.1016/j.ymthe.2024.06.025openalex-snapshot
Prospects and challenges of tissue-derived extracellular vesicles
inferred← citeshttps://doi.org/10.1021/acs.analchem.2c02378openalex-snapshot
Stability and Dissociation of Adeno-Associated Viral Capsids by Variable Temperature-Charge Detection-Mass Spectrometry
inferred← citeshttps://doi.org/10.1021/acs.analchem.2c04813openalex-snapshot
Using Peptide Nucleic Acid Hybridization Probes for Qualitative and Quantitative Analysis of Nucleic Acid Therapeutics by Capillary Electrophoresis
inferred← citeshttps://doi.org/10.1021/acs.analchem.3c02185openalex-snapshot
Combining Surface-Induced Dissociation and Charge Detection Mass Spectrometry to Reveal the Native Topology of Heterogeneous Protein Complexes
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.langmuir.2c02643openalex-snapshot
Empty and Full AAV Capsid Charge and Hydrophobicity Differences Measured with Single-Particle AFM
inferred← citeshttps://doi.org/10.1021/acsomega.2c01813openalex-snapshot
Electrophoresis-Mediated Characterization of Full and Empty Adeno-Associated Virus Capsids
inferred← citeshttps://doi.org/10.1021/cbmi.5c00070openalex-snapshot
An Automated Workflow Leveraging Machine Learning for Physical Titer Determination from Cryo-TEM Images of Adeno-Associated Virus Capsids
inferred← citeshttps://doi.org/10.1038/s41467-023-42439-4openalex-snapshot
Single virus fingerprinting by widefield interferometric defocus-enhanced mid-infrared photothermal microscopy
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The CRISPR Revolution in the Drug Discovery Workflow: An Industry Perspective
inferred← citeshttps://doi.org/10.1089/hum.2021.009openalex-snapshot
Characterization of Adeno-Associated Virus Capsid Proteins with Two Types of VP3-Related Components by Capillary Gel Electrophoresis and Mass Spectrometry
inferred← citeshttps://doi.org/10.1111/hae.14530openalex-snapshot
Gene therapy – are we ready now?
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Gene therapy for hemophilia: Current status and laboratory consequences
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What Went Wrong with VEGF-A in Peripheral Arterial Disease? A Systematic Review and Biological Insights on Future Therapeutics
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Regulatory Perspective for Biologics License Application of Recombinant Adeno-Associated Virus Products in China
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Manufacturing Cell and Gene Therapies: Challenges in Clinical Translation
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Advancing herbal medicine: enhancing product quality and safety through robust quality control practices
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The Top 100 Most Cited Articles on Platelet-Rich Plasma Use in Regenerative Medicine—A Bibliometric Analysis—From the ESSKA Orthobiologic Initiative
inferred← citeshttps://doi.org/10.3390/biomedicines14030653openalex-snapshot
An Improved Method for Determining the Infection Titer of Replication-Competent Adeno-Associated Virus
inferred← citeshttps://doi.org/10.3390/cimb46080499openalex-snapshot
Therapeutic Application and Structural Features of Adeno-Associated Virus Vector
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Biophysical Characterization of Adeno-Associated Virus Vectors Using Ion-Exchange Chromatography Coupled to Light Scattering Detectors
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Automated Mass Photometry of Adeno-Associated Virus Vectors from Crude Cell Extracts
inferred← citeshttps://doi.org/10.3390/molecules29225312openalex-snapshot
An In Vitro RNA Editing-Based Reporter Assay for Transcriptional Activity of Therapeutic Gene in Gene Therapy Products
inferred← citeshttps://doi.org/10.3390/molecules30102170openalex-snapshot
Methodological Study on Determination of Recombinant Adeno-Associated Virus Particle Titer Through Size Exclusion Chromatography with Multiangle Light Scattering and Collaborative Calibration of Standard Substances
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PCR-Based Analytical Methods for Quantification and Quality Control of Recombinant Adeno-Associated Viral Vector Preparations
inferred← citeshttps://doi.org/10.3390/pharmaceutics14010168openalex-snapshot
Report on Webinar Series Cell and Gene Therapy: From Concept to Clinical Use
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Backbone-Minimised Nanoplasmid DNA Systems Enable High-Titre AAV Production in Suspension HEK293 Platforms
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