ArticleMolecular therapy. Methods & clinical development2020
Vector Copy Distribution at a Single-Cell Level Enhances Analytical Characterization of Gene-Modified Cell Therapies.
Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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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
17 citing papers in PubMed, 26 citations in OpenAlex.
- Single-cell vector copy number analysis of phenotypically defined long-term hematopoietic stem cells for gene therapy safety assessment.Molecular therapy. Advances · 2026Article
- A single-cell multi-omics assay for simultaneous measurement of vector copy number and protein expression in CAR T cells.Molecular therapy. Advances · 2026Article
- Overcoming lentiviral delivery limitations in hard-to-transduce suspension cells for genome-wide CRISPR screening.Molecular therapy. Advances · 2026Article
- Adaptation of lentiviral vectors for viral gene therapy and their impact on host cell biology.Journal of translational medicine · 2026Review
- Site-specific genome engineering of primary human natural killer cells for programmable anti-tumor function.bioRxiv : the preprint server for biology · 2025Article
- Interlaboratory assessment of candidate reference materials for lentiviral vector copy number and integration site measurements.Molecular therapy. Methods & clinical development · 2025Article
- Establishing a GMP-compliant manufacturing process and phase-appropriate analytics for early development of a FiCAR T-cell product with a novel CAR spacer.Scientific reports · 2025Article
- An adjusted droplet digital PCR assay for quantification of vector copy number in CAR-T cell and TCR-T cell products.Immuno-oncology technology · 2025Article
- Review
- Forward programming of hiPSCs towards beta-like cells using Ngn3, Pdx1, and MafA.Scientific reports · 2024Article
- Increasing Gene Editing Efficiency via CRISPR/Cas9- or Cas12a-Mediated Knock-In in Primary Human T Cells.Biomedicines · 2024Review
- Release Assays and Potency Assays for CAR T-Cell Interventions.Advances in experimental medicine and biology · 2023Article
- Engineered T cells from induced pluripotent stem cells: from research towards clinical implementation.Frontiers in immunology · 2023Review
- Article
- Natural killer cells in clinical development as non-engineered, engineered, and combination therapies.Journal of hematology & oncology · 2022Review
- Targeted T cell receptor gene editing provides predictable T cell product function for immunotherapy.Cell reports. Medicine · 2021Article
- The Chimeric Antigen Receptor Detection Toolkit.Frontiers in immunology · 2020Review
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The ability to deliver transgenes into the human genome using viral vectors is a major enabler of the gene-modified cell therapy field. However, the control of viral transduction is difficult and can lead to product heterogeneity, impacting efficacy and safety, as well as increasing the risk of batch failure during manufacturing. To address this, we generated a novel analytical method to measure vector copy distribution at the single-cell level in a gene-modified, lentiviral-based immunotherapy model. As the limited amount of genomic DNA in a single cell hinders reliable quantification, we implemented a preamplification strategy on selected lentiviral and human gene targets in isolated live single cells, followed by quantification of amplified material by droplet digital PCR. Using a bespoke probability framework based on Bayesian statistics, we show that we can estimate vector copy number (VCN) integers with maximum likelihood scores. Notably, single-cell data are consistent with population analysis and also provide an overall measurement of transduction efficiency by discriminating transduced (VCN ≥ 1) from nontransduced (VCN = 0) cells. The ability to characterize cell-to-cell variability provides a powerful high-resolution approach for product characterization, which could ultimately allow improved control over product quality and safety.
Identifiers
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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.