ArticleHuman gene therapy2022
Insight and Development of Advanced Recombinant Adeno-Associated Virus Analysis Tools Exploiting Single-Particle Quantification by Multidimensional Droplet Digital PCR.
Article in Human gene therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed.
- Preparation and collaborative study of a recombinant adeno-associated virus (rAAV) genome copy number reference material.Journal of pharmaceutical analysis · 2026Article
- Development of high-throughput analytical methods for characterization and quantitation of rAAV genome integrity.Molecular therapy. Methods & clinical development · 2025Article
- Development of an HEK293 Suspension Cell Culture Medium, Transient Transfection Optimization Workflow, and Analytics for Batch rAAV Manufacturing.Biotechnology and bioengineering · 2025Article
- Impact of DNase digestion on titer measurements of engineered adeno-associated virus serotypes.Molecular therapy. Methods & clinical development · 2025Article
- Characterization of AAV vectors: A review of analytical techniques and critical quality attributes.Molecular therapy. Methods & clinical development · 2024Review
- Assessment of Key Factors Impacting Variability in AAV Vector Genome Titration by Digital PCR.International journal of molecular sciences · 2024Article
- Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System.Human gene therapy · 2024Article
- An improved high-resolution method for quantitative separation of empty and filled AAV8 capsids by strong anion exchange HPLC.Frontiers in bioengineering and biotechnology · 2024Article
- Design space determination to optimize DNA complexation and full capsid formation in transient rAAV manufacturing.Biotechnology and bioengineering · 2023Article
- Article
- Evaluation of the SH-SY5Y cell line as anFrontiers in molecular neuroscience · 2023Article
- Genome concentration, characterization, and integrity analysis of recombinant adeno-associated viral vectors using droplet digital PCR.PloS one · 2023Article
- Thermostability and in vivo performance of AAV9 in a film matrix.Communications medicine · 2022Article
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 become the most widely used vector in the gene therapy field with hundreds of clinical trials ongoing and already several products on the market. AAV's physicochemical stability, and the various natural and engineered serotypes allow for targeting a broad range of cell types and tissue by diverse routes of administration. Progressing from early clinical studies to eventual market approval, many critical quality attributes have to be defined and reproducibly quantified, such as AAV stability, purity, aggregates, empty/full particles ratio, and rAAV genome titration. Droplet digital PCR (ddPCR) is becoming the tool of choice to perform absolute quantification of rAAV genomes. In the present study, we have identified critical parameters that could impact AAV titration and characterization accuracy, such as Poisson distribution confidence interval, primers/probe position, and potential aggregates. Our work presents how ddPCR can help to better characterize AAV vectors on the single particle level and highlights challenges that we are facing today in terms of AAV titration.
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