LVV SMRTcap reveals extensive proviral variation in lentiviral vector-transduced CAR T cells

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Authors: C. J. Kaiser, Ghazal S. Mehs, Erin M Elliott, Joanna Jasinska Mroczkowska, Jain Ankita, Michael Ferguson, Alfred L. Garfall, Frederic D. Bushman, Joseph A. Fraietta, Eric Christian Rouchka, Melissa L. Smith
Cited by: 1
References: 48

Abstract

Lentiviral vectors are commonly used to introduce chimeric antigen receptor transgenes into T cells, but routine assays quantify vector copy number or integration sites without sequencing full-length integrated vectors. HIV-1 proviruses often acquire large deletions and cytidine deaminase-driven hypermutation; whether similar variation occurs in therapeutic lentiviral vectors is unclear. We adapted a novel long-read capture approach to enrich long fragments spanning vector DNA and adjacent human sequence, enabling simultaneous integration-site mapping and proviral integrity analysis with single-molecule resolution. In research-grade CAR T cells produced with an experimental, transient-transfection lentiviral vector workflow, 40% of integrated vectors carried recurrent deletions that removed the internal promoter or parts of the chimeric antigen receptor cassette. The dominant promoter deletion was present in the viral stock. In clinical chimeric antigen receptor T cell products, promoter deletions were less frequent, but detectable pre-infusion and post-infusion. Across datasets we observed widespread G-to-A substitutions consistent with restriction factor editing, including changes predicted to introduce premature stop codons within the transgene open reading frame. Our method reveals proviral variants invisible to standard quality-control assays and provides a framework to improve vector production and monitor transgene integrity in clinical products.

Fields of Study & Subjects

CAR-T cell therapy research Virus-based gene therapy research CRISPR and Genetic Engineering

Publication details

Journal / source
bioRxiv (Cold Spring Harbor Laboratory)
Publisher
openRxiv
Publication date
15 May 2026
Language
EN
Document type
Preprint