ArticleGene therapy2016
Measurement of lentiviral vector titre and copy number by cross-species duplex quantitative PCR.
Article in Gene therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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.
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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
19 citing papers in PubMed.
- Gene editing for Spinocerebellar ataxia type 3 taking advantage of the human ATXN3L paralog as replacement gene.Gene therapy · 2025Article
- Protocol to rapidly screen CRISPR-Cas9 gene editing outcomes in a cell population by mutating eGFP to a blue or non-fluorescent phenotype.STAR protocols · 2025Article
- Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia byCells · 2023Article
- Semi-automated workflows to quantify AAV transduction in various brain areas and predict gene editing outcome for neurological disorders.Molecular therapy. Methods & clinical development · 2023Article
- CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation.International journal of molecular sciences · 2022Article
- Self-driving armored CAR-T cells overcome a suppressive milieu and eradicate CD19Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- The impact of lentiviral vector genome size and producer cell genomic to gag-pol mRNA ratios on packaging efficiency and titre.Molecular therapy. Methods & clinical development · 2021Article
- Maximizing lentiviral vector gene transfer in the CNS.Gene therapy · 2021Article
- Application of droplet digital PCR for the detection of vector copy number in clinical CAR/TCR T cell products.Journal of translational medicine · 2020Article
- The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements.Journal of clinical medicine · 2019Article
- Overexpression of GATA2 Enhances Development and Maintenance of Human Embryonic Stem Cell-Derived Hematopoietic Stem Cell-like Progenitors.Stem cell reports · 2019Article
- Gene replacement therapy in a model of Charcot-Marie-Tooth 4C neuropathy.Brain : a journal of neurology · 2019Article
- GMP-Compliant Manufacturing of NKG2D CAR Memory T Cells Using CliniMACS Prodigy.Frontiers in immunology · 2019Article
- Short-hairpin RNA against aberrantHaematologica · 2018Article
- Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.Human gene therapy methods · 2018Article
- Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.Molecular therapy. Methods & clinical development · 2017Article
- Application of Droplet Digital PCR for Estimating Vector Copy Number States in Stem Cell Gene Therapy.Human gene therapy methods · 2016Article
- Lentiviral gene transfer into human and murine hematopoietic stem cells: size matters.BMC research notes · 2016Article
- Intrathecal gene therapy rescues a model of demyelinating peripheral neuropathy.Proceedings of the National Academy of Sciences of the United States of America · 2016Article
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Lentiviruses are the vectors of choice for many preclinical studies and clinical applications of gene therapy. Accurate measurement of biological vector titre before treatment is a prerequisite for vector dosing, and the calculation of vector integration sites per cell after treatment is as critical to the characterisation of modified cell products as it is to long-term follow-up and the assessment of risk and therapeutic efficiency in patients. These analyses are typically based on quantitative real-time PCR (qPCR), but as yet compromise accuracy and comparability between laboratories and experimental systems, the former by using separate simplex reactions for the detection of endogene and lentiviral sequences and the latter by designing different PCR assays for analyses in human cells and animal disease models. In this study, we validate in human and murine cells a qPCR system for the single-tube assessment of lentiviral vector copy numbers that is suitable for analyses in at least 33 different mammalian species, including human and other primates, mouse, pig, cat and domestic ruminants. The established assay combines the accuracy of single-tube quantitation by duplex qPCR with the convenience of one-off assay optimisation for cross-species analyses and with the direct comparability of lentiviral transduction efficiencies in different species.
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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.