ArticleMolecular therapy. Methods & clinical development2022
Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses.
Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
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Who cites it
40 citing papers in PubMed, 51 citations in OpenAlex.
- High resolution ES-DMA quantifies AAV capsid DNA content by electrical mobility to mass correlation.Gene therapy · 2026Article
- Modulating pressure in the Orbitrap improves sensitivity and mass resolution in charge detection mass spectrometry.Nature communications · 2026Article
- Construct optimization for AAV-mediated human α-syn overexpression, and validation across research settings: Development of a shared tool for the research community.Journal of Parkinson's disease · 2026Article
- Best practice mass photometry: a guide to optimal single-molecule mass measurement.Nature protocols · 2026Review
- Optimizing single molecule, real-time sequencing for enhanced characterization of adeno-associated viral vector genomes.Molecular therapy. Advances · 2026Article
- Analyses of Individual Singly Charged Ions Using a High-Field Orbitrap Analyzer.Analytical chemistry · 2026Article
- Imaged Capillary Isoelectric Focusing (icIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on their Transduction Efficiency.Current gene therapy · 2026Article
- Bridging science and hope: the evolving story of gene therapy for neuromuscular diseases.Frontiers in cell and developmental biology · 2026Review
- Integrated AAV optimization enables efficient gene delivery to kidney in murine and human tissue.Research square · 2025Article
- AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements.Molecular therapy. Methods & clinical development · 2025Article
- Breaking barriers in the sensitive and accurate mass determination of large DNA plasmids by mass photometry.Molecular therapy. Nucleic acids · 2025Article
- Mass photometry as a robust method for characterizing adeno-associated virus critical quality attributes in gene therapy vector.Molecular therapy. Nucleic acids · 2025Article
- Characterizing the Content and Structure of AAV Capsids by Size Exclusion Chromatography and Orbitrap-Based Charge Detection-Mass Spectrometry.Journal of the American Society for Mass Spectrometry · 2025Article
- Electron Capture-Induced Charge Reduction Benefits the Recording of Ultralong Transients in Orbitrap-Based Individual-Ion Mass Spectrometry.Analytical chemistry · 2025Article
- Mass Determination of Filled and Empty AAV5 Particles Enabled by Nanoelectrospray Ionization and Proton Transfer Charge Reduction.Analytical chemistry · 2025Article
- Prolonged Trapping of Adeno-Associated Virus Capsids Reveals that Genome Packaging Affects Single-Ion Mass Spectrometry Measurements.Journal of the American Chemical Society · 2025Article
- A Robust and Automated Platform for Charge Detection Mass Spectrometry of Megadalton Biotherapeutics.Analytical chemistry · 2025Article
- Rapid At-Line AAVX Affinity HPLC: Enabling Process Analytical Technology for Bioprocess Development of Adeno-Associated Virus Vectors.Biotechnology journal · 2025Article
- Multimass Analysis of Adeno-Associated Virus Vectors by Orbitrap-Based Charge Detection Mass Spectrometry.Analytical chemistry · 2024Article
- Probing recombinant AAV capsid integrity and genome release after thermal stress by mass photometry.Molecular therapy. Methods & clinical development · 2024Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adeno-associated viruses (AAVs) are useful vehicles for gene therapy because of their stability, low immunogenicity. and non-pathogenicity. However, disparity in AAV sample preparations (e.g., in capsid composition, DNA packaging, and impurities) gives rise to product heterogeneity, with possibly undesired effects on gene delivery. Ideally, AAV production should be with full control of AAV structure and genetic payload. Therefore, robust, efficient, and low material consuming methods are essential to characterize AAVs. Here, we use two emerging single-molecule techniques, mass photometry and Orbitrap-based charge-detection mass spectrometry, and show how they may efficiently and accurately characterize AAVs. We were able to resolve heterogeneous pools of particles, evaluating AAVs from two different serotypes (AAV8 and AAV2), produced by three independent production platforms, either lacking a genome or packed with a transgene. Together our data confirm that the different AAV production methods result in rather different and diverse AAV particle distributions. Especially for the packed AAVs, frequently additional subspecies were observed, next to the expected packed genome, mostly resulting from under- or overpackaging of genome material and/or residual empty particles. This work further establishes that both these single-particle techniques may become valuable tools in characterizing AAVs before they are used in gene therapy.
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