ArticleMolecular therapy. Methods & clinical development2021
Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry.
Article in Molecular therapy. Methods & clinical development, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers.
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Who cites it
60 citing papers in PubMed.
- 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
- Sedimentation velocity analytical ultracentrifugation (SV-AUC) for adeno-associated virus characterization: impact of cell alignment.European biophysics journal : EBJ · 2026Article
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Optimization and scale up strategies for reproducible AAV enrichment step on CIMmultusGene therapy · 2026Article
- DNA and RNA editing for the therapy of human diseases: current status, challenges, and future prospects.Molecular biomedicine · 2026Review
- Enhancer-based gene therapy: a new path for precision medicine.Hereditas · 2026Review
- Generation and characterization of a HEK293 cell line optimized for recombinant adeno-associated virus production.Biotechnology reports (Amsterdam, Netherlands) · 2026Article
- Article
- Development of high-throughput analytical methods for characterization and quantitation of rAAV genome integrity.Molecular therapy. Methods & clinical development · 2025Article
- Adeno-associated virus serotype 9 structural heterogeneity and stability characterized by charge detection mass spectrometry.Molecular therapy. Methods & clinical development · 2025Article
- HDX-MS reveals pH and temperature-responsive regions on AAV capsids and the structural basis for DNA release.Gene therapy · 2025Article
- ConSeqUMI, an error-free nanopore sequencing pipeline to identify and extract individual nucleic acid molecules from heterogeneous samples.Nucleic acids research · 2025Article
- Development and implementation of an LC-MS-based multi-attribute method for adeno-associated virus.Molecular therapy. Methods & clinical development · 2025Article
- AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements.Molecular therapy. Methods & clinical development · 2025Article
- Exploring AAV-Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Insights into Adeno-Associated Virus Capsid Charge Heterogeneity.Analytical chemistry · 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
- Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8.Molecular therapy. Methods & clinical development · 2025Article
- Tracking Particle-Encapsulated DNA Across the Anion-Exchange Chromatography Fractions of Recombinant Adeno-Associated Virus Using Droplet Digital PCR and High-Throughput Sequencing.Biotechnology journal · 2025Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recombinant adeno-associated virus (rAAV) has emerged as an important gene therapy vector with many clinical trials currently in progress. Analytical characterization and quantitation of particle content remain challenges in both the development and production of rAAV vectors. In this study, charge detection mass spectrometry (CDMS) and gel electrophoresis are used to characterize the DNA content of recombinant AAV8 (rAAV8) vectors with a wide range of target genome sizes. We show that the differences between the masses of empty particles and particles with the genome of interest (GOI) are correlated with the expected genome mass. A small systematic deviation (around 2%) is attributed to the packaging of counterions along with the DNA. In addition to the GOI, a broad distribution of heterogeneous DNA is packaged. The distribution peaks are close to the packaging capacity of the rAAV8 vectors. There is also evidence for the co-packaging of small DNA fragments along with the GOI. Finally, we present evidence that incubation at an elevated temperature can reduce the heterogeneity of the packaged DNA. Taken together, these results show that CDMS is a viable tool for characterization of the packaged genome.
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