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References from Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors. Local targets link to admitted publications; unresolved targets remain external evidence.
Adeno-associated virus capsid assembly is divergent and stochastic
10.1038/s41467-021-21935-5 · 2021 · External reference
Characterization of Adeno-Associated Virus Capsid Proteins with Two Types of VP3-Related Components by Capillary Gel Electrophoresis and Mass Spectrometry
2021 · External reference
Effect of Genome Size on AAV Vector Packaging
10.1038/mt.2009.255 · 2010 · External reference
Gene Therapy Using Adeno-Associated Virus Vectors
10.1128/cmr.00008-08 · 2008 · External reference
Engineering adeno-associated viruses for clinical gene therapy
10.1038/nrg3742 · 2014 · External reference
Cryo-electron Microscopy of Adeno-associated Virus
10.1021/acs.chemrev.1c00936 · 2022 · External reference
The Expression and Function of the Small Nonstructural Proteins of Adeno-Associated Viruses (AAVs)
10.3390/v16081215 · 2024 · External reference
Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings
2021 · External reference
The state of cell and gene therapy in 2023
10.1016/j.ymthe.2023.11.001 · 2023 · External reference
AAV vector development, back to the future
10.1016/j.ymthe.2025.03.064 · 2025 · External reference
Urgent call to action: Making gene therapy for Duchenne muscular dystrophy (DMD) affordable and accessible
10.1111/bcp.15900 · 2023 · External reference
Viral vector-based gene therapies in the clinic
10.1002/btm2.10258 · 2022 · External reference
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
10.3389/fimmu.2022.1001263 · 2022 · External reference
Characterization of AAV vectors: A review of analytical techniques and critical quality attributes
10.1016/j.omtm.2024.101309 · 2024 · External reference
Process and quality considerations for recombinant adeno-associated virus manufacturing platforms
10.1016/j.tibtech.2025.02.016 · 2025 · External reference
Towards a Platform Chromatography Purification Process for Adeno-Associated Virus (AAV)
10.1002/biot.202400526 · 2025 · External reference
Chromatography in Downstream Processing of Recombinant Adeno-Associated Viruses: A Review of Current and Future Practises
10.1002/bit.28932 · 2025 · External reference
Downstream processing of AAV based gene therapy vectors
10.1016/j.seppur.2025.133051 · 2025 · External reference
Development of an HEK293 Suspension Cell Culture Medium, Transient Transfection Optimization Workflow, and Analytics for Batch rAAV Manufacturing
10.1002/bit.28980 · 2025 · External reference
Improved productivity of recombinant adeno-associated virus (rAAV) via triple transfection of HEK293 cells using perfusion cultivation
10.1007/s00449-025-03167-9 · 2025 · External reference
Significant differences in capsid properties and potency between AAV vectors produced in Sf9 and HEK293 cells
10.1089/hum.2022.116 · 2023 · External reference
Systematic comparison of rAAV vectors manufactured using large-scale suspension cultures of Sf9 and HEK293 cells
10.1016/j.ymthe.2023.11.022 · 2024 · External reference
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors
10.1016/j.omtm.2020.05.018 · 2020 · External reference
Scalable serum-free production of recombinant adeno-associated virus type 2 by transfection of 293 suspension cells
10.1016/j.jviromet.2007.03.014 · 2007 · External reference
Production of adeno-associated virus (AAV) serotypes by transient transfection of HEK293 cell suspension cultures for gene delivery
10.1016/j.jviromet.2013.10.038 · 2014 · External reference
Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the CultureMedia for GMP FIX and FLT1 Clinical Vector
10.1038/mt.2015.187 · 2016 · External reference
The state of technological advancement to address challenges in the manufacture of rAAV gene therapies
10.1016/j.biotechadv.2024.108433 · 2024 · External reference
Rational downstream development for adeno-associated virus full/empty capsid separation – A streamlined methodology based on high-throughput screening and mechanistic modeling
10.1016/j.chroma.2024.464632 · 2024 · External reference
Combining steric exclusion with anion exchange – development of a universal and scalable adeno-associated virus downstream process
10.1016/j.chroma.2025.465674 · 2025 · External reference
Consecutive Affinity and Ion-Exchange Chromatography for AAV9 Vectors Purification
10.3390/biomedicines13020361 · 2025 · External reference
Improved adeno-associated virus empty and full capsid separation using weak partitioning multi-column AEX chromatography
10.1002/biot.202300245 · 2024 · External reference
Purification of AAV8 through a scalable two-step monolithic chromatography approach
10.1016/j.chroma.2024.465586 · 2025 · External reference
High-yield recombinant adeno-associated viral vector production by multivariate optimization of bioprocess and transfection conditions
10.1002/btpr.3445 · 2024 · External reference
Tuning capsid formation dynamics in recombinant adeno-associated virus producing synthetic cell lines to enhance full particle productivity
10.1002/biot.202400051 · 2024 · External reference
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform
10.1016/j.omtm.2024.101320 · 2024 · External reference
Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences
10.1016/j.omtm.2021.12.006 · 2022 · External reference
Establishment of a novel human amniotic epithelial-derived cell line, HAT, for high-yield AAV vector production
10.1016/j.omtm.2025.101594 · 2025 · External reference
Creation of a High-Yield AAV Vector Production Platform in Suspension Cells Using a Design-of-Experiment Approach
10.1016/j.omtm.2020.06.004 · 2020 · External reference
Design space determination to optimize DNA complexation and full capsid formation in transient rAAV manufacturing
10.1002/bit.28508 · 2023 · External reference
Enhancing the production of adeno-associated virus (AAV)2 and AAV9 with high full capsid ratio in HEK293 cells through design-of-experiment optimization of triple plasmid ratio
10.1002/biot.202300667 · 2024 · External reference
Unlocking DOE potential by selectingthe most appropriate design for rAAV optimization
10.1016/j.omtm.2024.101329 · 2024 · External reference
Direct Identification and Quantification of Recombinant Adeno- Associated Virus in Crude Cell Lysate and Conditioned Medium by Mass Photometry
10.1021/acs.analchem.5c00793 · 2025 · External reference
Variation of VP2 stoichiometry and deamidation of VP1 during production and their impacts on the transduction efficiency of AAV vectors
10.1016/j.omtm.2025.101581 · 2025 · External reference
Combined 100 keV cryo-electron microscopy and image analysis methods to characterize the wider adeno-associated viral products
10.1016/j.xphs.2024.03.026 · 2024 · External reference
Enhancement of recombinant adeno-associatedvirus activity by improved stoichiometry and homogeneity of capsid protein assembly
10.1016/j.omtm.2023.101142 · 2023 · External reference
Use of cesium chloride density gradient ultracentrifugation for the purification and characterization of recombinant adeno-associated virus
10.1007/s00249-025-01751-1 · 2025 · External reference
Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function
10.1016/j.ymthe.2018.09.013 · 2018 · External reference
Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea
10.1089/hum.2020.070 · 2020 · External reference
Differential T cell immune responses to deamidated adeno-associated virus vector
10.1016/j.omtm.2022.01.005 · 2022 · External reference
Focused Differential Glycan Analysis with the Platform Antibody-assisted Lectin Profiling for Glycan-related Biomarker Verification
10.1074/mcp.m800308-mcp200 · 2009 · External reference
Glycosylation of recombinant adeno-associated virus serotype 6
10.1016/j.omtm.2024.101256 · 2024 · External reference
Analytical Dissection of Minor Glycoforms and Glycoprotein Associations in rAAV Preparations by Multimodal Glycoproteomics
10.1007/s00216-025-06042-4 · 2025 · External reference
Lethal immunotoxicityin high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · 2023 · External reference
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy
10.1056/nejmoa2307798 · 2023 · External reference
Empty virions in AAV8 vector preparations reduce transduction efficiency and may cause total viral particle dose-limiting side effects
10.1038/mtm.2013.9 · 2014 · External reference
Comparison of Different Liquid Chromatography-Based Purification Strategies for Adeno-Associated Virus Vectors
10.3390/pharmaceutics13050748 · 2021 · External reference
Manufacturing challenges and rational formulation development for AAV viral vectors
10.1016/j.xphs.2021.03.024 · 2021 · External reference
Unraveling Cytotoxicity in HEK293 Cells During Recombinant AAV Production for Gene Therapy Applications
10.1002/biot.202400501 · 2025 · External reference
Adeno-associated virus perfusion enhanced expression: A commercially scalable, hightiter, high quality producer cell line process
10.1016/j.omtm.2024.101266 · 2024 · External reference
Multi-omics kinetic analysis of recombinant adeno-associated virus production by plasmid transfection of HEK293 cells
10.1002/btpr.3428 · 2024 · External reference
Comparative transcriptomic and proteomic kinetic analysis of adeno-associated virus production systems
10.1007/s00253-024-13203-5 · 2024 · External reference
Adeno-associated virus vector system controlling capsid expression improves viral quantity and quality
10.1016/j.isci.2023.106487 · 2023 · External reference
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8
10.1016/j.omtm.2025.101480 · 2025 · External reference
Size Distribution Analysis of the Adeno-Associated Virus Vector by the c(s) Analysis of Band Sedimentation Analytical Ultracentrifugation with Multiwavelength Detection
10.1016/j.xphs.2022.10.023 · 2023 · External reference
Sedimentation Analysis of Noninteracting and Self-Associating Solutes Using Numerical Solutions to the Lamm Equation
10.1016/s0006-3495(98)74069-x · 1998 · External reference
Computer-aided interpretation of analytical sedimentation data for proteins
1992 · External reference
Comprehensive Size Distribution and Composition Analysis of Adeno-Associated Virus Vector by Multiwavelength Sedimentation Velocity Analytical Ultracentrifugation
10.1016/j.xphs.2021.06.031 · 2021 · External reference
Multimass Analysis of Adeno-Associated Virus Vectors by Orbitrap-Based Charge Detection Mass Spectrometry
10.1021/acs.analchem.4c05229 · 2024 · External reference
Recombinant AAV batch profiling by nanoporesequencing elucidates product-related DNAimpurities and vector genome length distribution
10.1016/j.omtm.2025.101417 · 2025 · External reference
A novel protocol for in-depth analysis of recombinant adeno-associated virus capsid proteins using UHPLC–MS/MS
10.1002/rcm.9247 · 2022 · External reference
An AsCas12f-based compact genome-editing tool derived by deep mutational scanning and structural analysis
10.1016/j.cell.2023.08.031 · 2023 · External reference