ArticleHuman gene therapy methods2019
Lot-to-Lot Variation in Adeno-Associated Virus Serotype 9 (AAV9) Preparations.
Article in Human gene therapy methods, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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
15 citing papers in PubMed, 21 citations in OpenAlex.
- Intrathecal Delivery of Macromolecules to the Spinal Cord Enabled by Laser-Activated Perfluorocarbon Nanodroplets.ACS nano medicine · 2026Article
- Seroprevalence of anti-AAV antibodies in a healthy adult Spanish population: findings from the SAAVIA study.Gene therapy · 2026Article
- Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects.Biomedicines · 2024Review
- Use of adeno-associated viruses for transgenic modulation of microglia structure and function: A review of technical considerations and challenges.Brain, behavior, and immunity · 2024Review
- Single-Ion Mass Spectrometry for Heterogeneous and High Molecular Weight Samples.Journal of the American Chemical Society · 2024Review
- Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System.Human gene therapy · 2024Article
- Addgene AAV Data Hub: a platform for sharing AAV experimental data.Nature methods · 2023Article
- Orbitrap-Based Mass and Charge Analysis of Single Molecules.Accounts of chemical research · 2023Article
- Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard.Molecular therapy. Methods & clinical development · 2023Article
- Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses.Molecular therapy. Methods & clinical development · 2022Article
- Effects of Molecular Size on Resolution in Charge Detection Mass Spectrometry.Analytical chemistry · 2022Article
- Article
- Approaches to Heterogeneity in Native Mass Spectrometry.Chemical reviews · 2022Review
- Article
- Chemogenetics: Beyond Lesions and Electrodes.Neurosurgery · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
Viral vectors are complex drugs that pose a particular challenge for manufacturing. Previous studies have shown that, unlike small-molecule drugs, vector preparations do not yield a collection of identical particles. Instead, a mixture of particles that vary in capsid stoichiometry and impurities is created, which may differ from lot to lot. The consequences of this are unclear, but conflicting reports regarding the biological properties of vectors, including transduction patterns, suggest that this variability may have an effect. However, other variables, including differences in animal strains and techniques, make it difficult to identify a cause. Here, we report lot-to-lot variation in spinal cord gray matter transduction following intrathecal delivery of self-complementary adeno-associated virus serotype 9 vectors. Eleven lots of vector were evaluated from six vector cores, including one preclinical/Good Laboratory Practice lot. Eight of the lots, including the preclinical lot, failed to transduce the gray matter, whereas the other three provided robust transduction. The cause for this variation is unknown, but it did not correlate with vector titer, buffer, or purification method. These results highlight the need to identify the cause of this variation and to develop improved production and quality control methods to ensure lot-to-lot consistency of vector potency.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.