Article

HTLV-1 Splice Sites in Prevalent Gene Vectors Cause Splicing Perturbations in Transgenic Human Cells

2023-01-30

Abstract excerpt

<h4>ABSTRACT</h4> The use of any semi-randomly integrating gene vector in a therapeutic setting is associated with genotoxic risks. The two major mechanisms of genotoxicity are disruption of a coding sequence (loss-of-function) or transcriptional upregulation of genes (gain-of-function) in the cellular genome where the genetic modifications are executed. A third, less widely recognized genotoxic risk stems from s...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: b4acaec7-6ac3-5dea-903e-d7862d6502b8
DOI: 10.1101/2023.01.28.526022

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

HTLV-1 Splice Sites in Prevalent Gene Vectors Cause Splicing Perturbations in Transgenic Human CellsDOI 10.1101/2023.01.28.526022Read

Current landscape of vector safety and genotoxicity after hematopoietic stem or immune cell gene therapySemantic proximity Read

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral VectorsSemantic proximity Read

Risk and Benefit Assessment of Gene Therapy with Lentiviral Vectors and Hematopoietic Stem Cells: The Skysona CaseSemantic proximity Read

Safety of Gene Therapy: New Insights to a Puzzling CaseSemantic proximity Read

Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells.Semantic proximity Read

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy.Semantic proximity Read

Aberrant splicing events caused by insertion of genes of interest into expression vectors.Semantic proximity Read

Ex vivo long-term expansion of human hematopoietic stem and progenitor cells as a tool for modeling vector integration sites and clonalitySemantic proximity Read

Retroelements in human disease.Semantic proximity Read

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis.Semantic proximity Read

Specific targeted gene repair using single-stranded DNA oligonucleotides at an endogenous locus in mammalian cells uses homologous recombination.Semantic proximity Read

hInGeTox: A human-based in vitro platform to evaluate lentivirus contribution to genotoxicitySemantic proximity Read