Evidence map›Paper›PMID 40621474›Full record

ArticleMolecular therapy. Methods & clinical development2025

Interlaboratory assessment of candidate reference materials for lentiviral vector copy number and integration site measurements.

Hua-Jun He, Zhiyong He, Steven P Lund, Laure Turner, Yongjun Fan, Yu Qiu, David C Corney, Boro Dropulic, Rimas Orentas, Oxana Slessareva and 33 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

43 authors.

Hua-Jun HeNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Zhiyong HeNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Steven P LundNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Laure TurnerGENEWIZ from Azenta Life Sciences, 111 Corporate Boulevard, South Plainfield, NJ 07080, USA.
Yongjun FanGENEWIZ from Azenta Life Sciences, 111 Corporate Boulevard, South Plainfield, NJ 07080, USA.
Yu QiuGENEWIZ from Azenta Life Sciences, 111 Corporate Boulevard, South Plainfield, NJ 07080, USA.
David C CorneyGENEWIZ from Azenta Life Sciences, 111 Corporate Boulevard, South Plainfield, NJ 07080, USA.
Boro DropulicCaring Cross, 910 Clopper Road, Gaithersburg, MD 20878, USA.
Rimas OrentasCaring Cross, 910 Clopper Road, Gaithersburg, MD 20878, USA.
Oxana SlessarevaCaring Cross, 910 Clopper Road, Gaithersburg, MD 20878, USA.
Priscilla WelchCastle Creek Biosci, 405 Eagleview Boulevard, Exton, PA 19341, USA.
Katie DungcaCastle Creek Biosci, 405 Eagleview Boulevard, Exton, PA 19341, USA.
Ellen StellooCergentis, a Solvias Company, Yalelaan 62, 3584 CM Utrecht, the Netherlands.
Gabrielle DijksteelCergentis, a Solvias Company, Yalelaan 62, 3584 CM Utrecht, the Netherlands.
Harma FeitsmaCergentis, a Solvias Company, Yalelaan 62, 3584 CM Utrecht, the Netherlands.
Sana Ahmed-SeghirGenomic Vision, 80 Rue des Meuniers, Bagneux, Paris Area 92220, France.
Rostyslav MakarenkoGenomic Vision, 80 Rue des Meuniers, Bagneux, Paris Area 92220, France.
Engin AltunluGenomic Vision, 80 Rue des Meuniers, Bagneux, Paris Area 92220, France.
Daniëlle SteenmansGlycostem Therapeutics, Kloosterstraat 9, 5349 AB Oss, the Netherlands.
Jan SpanholtzGlycostem Therapeutics, Kloosterstraat 9, 5349 AB Oss, the Netherlands.
Monica RaimoGlycostem Therapeutics, Kloosterstraat 9, 5349 AB Oss, the Netherlands.
Shai SenderovichGSK, Gunnels Wood Road, Stevenage SG1 2NF, UK.
Barbara S PaughLentigen, a Miltenyi Biotec Company, 1201 Clopper Road, Gaithersburg, MD 20878, USA.
Chieh-Yuan LiMission Bio, 400 E Jamie Court, South San Francisco, CA 94080, USA.
Benjamin SchroederMission Bio, 400 E Jamie Court, South San Francisco, CA 94080, USA.
Alexandra S WhaleNational Measurement Laboratory (NML at LGC), LGC, Queens Road, Teddington, Middlesex TW11 0LY, UK.
Dilek YenerNational Measurement Laboratory (NML at LGC), LGC, Queens Road, Teddington, Middlesex TW11 0LY, UK.
Carole A FoyNational Measurement Laboratory (NML at LGC), LGC, Queens Road, Teddington, Middlesex TW11 0LY, UK.
Shareef NahasSampled, 30 Knightsbridge Road, Piscataway, NJ 08854, USA.
Feng TuSampled, 30 Knightsbridge Road, Piscataway, NJ 08854, USA.
Michael SheldonSampled, 30 Knightsbridge Road, Piscataway, NJ 08854, USA.
Yan DingSampled, 30 Knightsbridge Road, Piscataway, NJ 08854, USA.
Jennifer KandellThermo Fisher Scientific, 10421 Wateridge Circle, San Diego, CA 92121, USA.
Uma LakshmipathyThermo Fisher Scientific, 10421 Wateridge Circle, San Diego, CA 92121, USA.
Jennifer H McDanielNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Justin M ZookNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Sierra MillerNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Samantha MaraghNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Simona PatangeNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Mahir MohiuddinNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Alessandro TonaNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Kenneth D ColeNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.
Sheng Lin-GibsonNational Institute of Standards and Technology, 100 Bureau Drive, Gaithersburg, MD 20899, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

While lentiviral vectors have played a critical role in the emergence of gene-modified cell therapies, safety concerns remain regarding potential insertional mutagenesis. Regulatory authorities strongly recommend risk assessment and management of vector copy numbers (VCNs), integration profiles, and integration sites in the lentivirus-based cell and gene therapy products. However, accurately measuring these parameters remains a significant challenge due to the lack of standardized methodologies and VCN reference materials (RMs). Toward this challenge, we conducted an interlaboratory study on NIST candidate RMs for VCN measurements. The candidate RMs comprise five human genomic DNA samples or fixed cells from clonal Jurkat cell lines with defined VCNs ranging from 0 to 4. All 12 study participants were able to identify the VCN in the five blinded samples using quantitative PCR (qPCR), digital PCR (dPCR), or next generation sequencing (NGS) assays. Consensus value of VCN and integration sites in these candidate RMs were achieved. The fixed clonal VCN cells were also used to evaluate an emerging imaging-based technology called molecular combing. This interlaboratory assessment demonstrated the utility, commutability, and suitability of the NIST VCN candidate RMs for quality assurance and improved confidence in VCN, integration profile, and integration site measurements.

Indexed as

CAR T-cell therapydigital PCRgene therapyintegration profilingintegration site analysisinterlaboratory testinglentiviral vector, LVnext generation sequencingNGSreference materialvector copy number, VCN

Identifiers

PMID40621474
PMCPMC12229725

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Registered trials

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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.