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April 1, 2026Human Gene Therapy0 citations

AAV Vectors Have Limited Impact on Host Chromatin Accessibility and Nuclear Architecture

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JZJoël ZölligMPMaija K. PietiläMBMelina Bräuer

Key Points

  • The aim is to assess how recombinant adeno-associated virus (AAV) vectors affect host chromatin remodeling and nuclear organization.
  • Evaluated human primary fibroblast and lung carcinoma cells after infection with different AAV vectors.
  • Used genome-wide assay for transposase-accessible chromatin sequencing to measure chromatin accessibility.
  • Conducted DNase I digestion assays on specific genomic loci to support findings.
  • Performed immunofluorescence-based single-cell analyses to observe nuclear morphology and polymerase signals.
  • No detectable changes in host chromatin accessibility were observed at either 24 or 48 hours.
  • Mild changes in histone abundance and RNA polymerase II signals were noted in some cases.
  • Nuclear morphology and organization of nuclear compartments showed modest changes, especially influenced by vector type and cell state.

Abstract

Recombinant adeno-associated virus (rAAV) vectors are widely used for gene therapeutics, yet their early effects on host chromatin remodeling and nuclear organization remain insufficiently characterized. In contrast, several DNA viruses are known to remodel host chromatin accessibility, for example, Herpes simplex virus type 1 (HSV-1) in mammalian cells and baculoviruses in insect systems. Here, we evaluated genome accessibility and nuclear organization of primary human fibroblast cells at 24 and 48 h after infection with wild-type AAV2, single-stranded (ss) and self-complementary rAAV2, as well as ssAAV2 and ssAAV-DJ in human lung carcinoma cells. Genome-wide assay for transposase-accessible chromatin using sequencing showed no detectable change in host chromatin accessibility at either time point. A DNase I digestion assay at five candidate loci supported this observation. Although host accessibility remained stable, immunofluorescence-based single-cell analyses uncovered modest changes in histone abundance, RNA polymerase II–associated signals, nuclear morphology, and the organization of liquid–liquid phase-separated nuclear compartments. These effects were generally mild in primary fibroblasts but showed greater dependence on vector type, cell cycle state, and transgene expression in carcinoma cells. Collectively, at the tested doses and times, AAV-based vectors did not remodel chromatin accessibility at scale and induced only small changes in polymerase-associated readouts and nuclear architecture. These data are consistent with limited nuclear perturbation under these conditions.

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Cite This Study

Zöllig et al. (2026) studied this question.

synapsesocial.com/papers/69cd7e935652765b073a9950https://doi.org/10.1177/10430342261437975
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