ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Stem cell marking with promotor-deprived self-inactivating retroviral vectors does not lead to induced clonal imbalance.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 31 citations in OpenAlex.
- Clonal competition in BcrAbl-driven leukemia: how transplantations can accelerate clonal conversion.Molecular cancer · 2017Article
- Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015Review
- Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model.Molecular therapy. Nucleic acids · 2014Article
- Lentiviral and targeted cellular barcoding reveals ongoing clonal dynamics of cell lines in vitro and in vivo.Genome biology · 2014Article
- Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes.Gene therapy · 2013Article
- From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.Human gene therapy. Clinical development · 2013Article
- Biosafety features of lentiviral vectors.Human gene therapy · 2013Review
- Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Evaluation of residual promoter activity in γ-retroviral self-inactivating (SIN) vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Gene therapy strategies: can we eradicate HIV?Current HIV/AIDS reports · 2011Article
- RGB marking facilitates multicolor clonal cell tracking.Nature medicine · 2011Article
- Article
- SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Recent advances in lentiviral vector development and applications.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Review
- Gene therapy activates EVI1, destabilizes chromosomes.Nature medicine · 2010Article
- Self-inactivating retroviral vector-mediated gene transfer induces oncogene activation and immortalization of primary murine bone marrow cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Basic research tries to decrease the risks of translational medicine.Gene therapy · 2009Article
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Stable genetic modification of stem cells holds great promise for gene therapy and marking, but commonly used gamma-retroviral vectors were found to influence growth/survival characteristics of hematopoietic stem cells (HSCs) by insertional mutagenesis. In this article, we show that promoter-deprived gamma-retroviral self-inactivating (pd-SIN) vectors allow stable genetic marking of serially reconstituting murine HSC. In contrast to findings with gamma-retroviral long terminal repeat (LTR) vectors, serial transplantation of pd-SIN-marked HSC in a sensitive mouse model was apparently not associated with induced clonal imbalance of gene-marked HSC. Furthermore, insertions of pd-SIN into protooncogenes, growth-promoting and signaling genes occurred significantly less frequent than in control experiments with LTR vectors. Also, transcriptional dysregulation of neighboring genes potentially caused by the pd-SIN insertion was rarely seen and comparatively weak. The integration pattern of promotor-deprived SIN vectors in reconstituting HSC seems to depend on the transcriptional activity of the respective gene loci reflecting the picture described for LTR vectors. In conclusion, our data strongly support the use of SIN vectors for gene-marking studies and suggest an increased therapeutic index for vectors lacking enhancers active in HSC.
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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.