ArticleMolecular therapy : the journal of the American Society of Gene Therapy2011
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
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Who cites it
32 citing papers in PubMed.
- Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.The Journal of clinical investigation · 2017Trial
- Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.Science (New York, N.Y.) · 2013Trial
- A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.Nature communications · 2024Article
- Vascular endothelial growth factor a modified mRNA engineered cellular electrospun membrane complexes promotes mouse skin wound repair.Materials today. Bio · 2023Article
- Evolution of Gene Therapy, Historical Perspective.Hematology/oncology clinics of North America · 2022Review
- Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34Haematologica · 2022Article
- Retroviral gene therapy in Germany with a view on previous experience and future perspectives.Gene therapy · 2021Review
- Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders.Open life sciences · 2021Review
- Gene Therapy of the Hemoglobinopathies.HemaSphere · 2020Review
- Interactions between Retroviruses and the Host Cell Genome.Molecular therapy. Methods & clinical development · 2018Review
- Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE.Molecular therapy. Methods & clinical development · 2018Article
- Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Genome-wide Mapping of Off-Target Events in Single-Stranded Oligodeoxynucleotide-Mediated Gene Repair Experiments.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Retargeted Foamy Virus Vectors Integrate Less Frequently Near Proto-oncogenes.Scientific reports · 2016Article
- Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- A Graph Based Framework to Model Virus Integration Sites.Computational and structural biotechnology journal · 2016Article
- Gene therapy returns to centre stage.Nature · 2015Review
- Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model.Molecular therapy. Nucleic acids · 2014Article
- Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice.Human gene therapy · 2014Article
- Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.Human gene therapy · 2014Review
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29 authors.
Funding
Abstract
Vector-associated side effects in clinical gene therapy have provided insights into the molecular mechanisms of hematopoietic regulation in vivo. Surprisingly, many retrovirus insertion sites (RIS) present in engrafted cells have been found to cluster nonrandomly in close association with specific genes. Our data demonstrate that these genes directly influence the in vivo fate of hematopoietic cell clones. Analysis of insertions thus far has been limited to individual clinical studies. Here, we studied >7,000 insertions retrieved from various studies. More than 40% of all insertions found in engrafted gene-modified cells were clustered in the same genomic areas covering only 0.36% of the genome. Gene classification analyses displayed significant overrepresentation of genes associated with hematopoietic functions and relevance for cell growth and survival in vivo. The similarity of insertion distributions indicates that vector insertions in repopulating cells cluster in predictable patterns. Thus, insertion analyses of preclinical in vitro and murine in vivo studies as well as vector insertion repertoires in clinical trials yielded concerted results and mark a small number of interesting genomic loci and genes that warrants further investigation of the biological consequences of vector insertions.
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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.