ArticleHuman gene therapy2014
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.
Article in Human gene therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers, 1 of them a synthesis that pooled it.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
59 citing papers in PubMed, 1 synthesis or guideline pooled it.
- What's new and what's next for gene therapy in Pompe disease?Expert opinion on biological therapy · 2022Pooled it
- Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.Blood · 2018Trial
- It's what's on the inside that counts: Capsid content as a critical quality attribute for AAV characterization.Molecular therapy. Advances · 2026Article
- An Improved Method for Determining the Infection Titer of Replication-Competent Adeno-Associated Virus.Biomedicines · 2026Article
- From molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.Frontiers in pharmacology · 2026Review
- Article
- Self-silencing adenovirus enables precise infectious titration of recombinant adeno-associated viral vectors.Molecular therapy. Methods & clinical development · 2025Article
- Review
- An SDS-NaOH-based method to isolate genome of recombinant adeno-associated virus vectors for physical titer measurement.PloS one · 2025Article
- Characterization of residual microRNAs in AAV vector batches produced in HEK293 mammalian cells and Sf9 insect cells.Molecular therapy. Methods & clinical development · 2024Article
- Article
- Article
- Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System.Human gene therapy · 2024Article
- Microfluidic AAV Purity Characterization: New Insights into Serotype and Sample Treatment Variability.ACS omega · 2024Article
- PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development.Molecular therapy. Methods & clinical development · 2023Review
- Partial genome content within rAAVs impacts performance in a cell assay-dependent manner.Molecular therapy. Methods & clinical development · 2023Article
- Transcriptomic Analysis Reveals the Inability of Recombinant AAV8 to Activate Human Monocyte-Derived Dendritic Cells.International journal of molecular sciences · 2023Article
- AAV2/9-mediated gene transfer into murine lacrimal gland leads to a long-term targeted tear film modification.Molecular therapy. Methods & clinical development · 2022Article
- Thermostability and in vivo performance of AAV9 in a film matrix.Communications medicine · 2022Article
- Insight and Development of Advanced Recombinant Adeno-Associated Virus Analysis Tools Exploiting Single-Particle Quantification by Multidimensional Droplet Digital PCR.Human gene therapy · 2022Article
Corrections and comments
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Authors and funding
36 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Abstract Gene therapy approaches using recombinant adeno-associated virus serotype 2 (rAAV2) and serotype 8 (rAAV8) have achieved significant clinical benefits. The generation of rAAV Reference Standard Materials (RSM) is key to providing points of reference for particle titer, vector genome titer, and infectious titer for gene transfer vectors. Following the example of the rAAV2RSM, here we have generated and characterized a novel RSM based on rAAV serotype 8. The rAAV8RSM was produced using transient transfection, and the purification was based on density gradient ultracentrifugation. The rAAV8RSM was distributed for characterization along with standard assay protocols to 16 laboratories worldwide. Mean titers and 95% confidence intervals were determined for capsid particles (mean, 5.50×10(11) pt/ml; CI, 4.26×10(11) to 6.75×10(11) pt/ml), vector genomes (mean, 5.75×10(11) vg/ml; CI, 3.05×10(11) to 1.09×10(12) vg/ml), and infectious units (mean, 1.26×10(9) IU/ml; CI, 6.46×10(8) to 2.51×10(9) IU/ml). Notably, there was a significant degree of variation between institutions for each assay despite the relatively tight correlation of assay results within an institution. This outcome emphasizes the need to use RSMs to calibrate the titers of rAAV vectors in preclinical and clinical studies at a time when the field is maturing rapidly. The rAAV8RSM has been deposited at the American Type Culture Collection (VR-1816) and is available to the scientific community.
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