ArticleNucleic acids research2007
An S/MAR-based infectious episomal genomic DNA expression vector provides long-term regulated functional complementation of LDLR deficiency.
Article in Nucleic acids research, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 55 citations in OpenAlex.
- Non-replicative herpes simplex virus genomic and amplicon vectors for gene therapy - an update.Gene therapy · 2025Review
- An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny.Stem cell reports · 2022Article
- Episomal minicircles persist in periods of transcriptional inactivity and can be transmitted through somatic cell nuclear transfer into bovine embryos.Molecular biology reports · 2019Article
- Lipid-mRNA Nanoparticle Designed to Enhance Intracellular Delivery Mediated by Shock Waves.ACS applied materials & interfaces · 2019Article
- The IS2 Element Improves Transcription Efficiency of Integration-Deficient Lentiviral Vector Episomes.Molecular therapy. Nucleic acids · 2018Article
- Episomal Nonviral Gene Therapy Vectors Slow Progression of Atherosclerosis in a Model of Familial Hypercholesterolemia.Molecular therapy. Nucleic acids · 2016Article
- The infectious BAC genomic DNA expression library: a high capacity vector system for functional genomics.Scientific reports · 2016Article
- Restoration of Physiologically Responsive Low-Density Lipoprotein Receptor-Mediated Endocytosis in Genetically Deficient Induced Pluripotent Stem Cells.Scientific reports · 2015Article
- Expanded GAA repeats impair FXN gene expression and reposition the FXN locus to the nuclear lamina in single cells.Human molecular genetics · 2015Article
- A GAA repeat expansion reporter model of Friedreich's ataxia recapitulates the genomic context and allows rapid screening of therapeutic compounds.Human molecular genetics · 2013Article
- Genetic modification of dividing cells using episomally maintained S/MAR DNA vectors.Molecular therapy. Nucleic acids · 2013Article
- RNAi-mediated knockdown of HMG CoA reductase enhances gene expression from physiologically regulated low-density lipoprotein receptor therapeutic vectors in vivo.Gene therapy · 2012Article
- Development of safer gene delivery systems to minimize the risk of insertional mutagenesis-related malignancies: a critical issue for the field of gene therapy.ISRN oncology · 2012Article
- Genetic modification of cancer cells using non-viral, episomal S/MAR vectors for in vivo tumour modelling.PloS one · 2012Article
- LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives.International archives of medicine · 2010Article
- Stable S/MAR-based episomal vectors are regulated at the chromatin level.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2010Article
- Herpes simplex virus type 1/adeno-associated virus hybrid vectors.The open virology journal · 2010Article
- Long-term physiologically regulated expression of the low-density lipoprotein receptor in vivo using genomic DNA mini-gene constructs.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Herpes Virus Amplicon Vectors.Viruses · 2009Article
- Physiological transgene regulation and functional complementation of a neurological disease gene deficiency in neurons.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
Corrections and comments
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Authors and funding
3 authors at 3 institutions in 2 countries.
Funding
Abstract
Episomal gene expression vectors offer a safe and attractive alternative to integrating vectors. Here we describe the development of a high capacity episomal vector system exploiting human episomal retention sequences to provide efficient vector maintenance and regulated gene expression through the delivery of a genomic DNA locus. The iBAC-S/MAR vector is capable of the infectious delivery and retention of large genomic DNA transgenes by exploiting the high transgene capacity of herpes simplex virus type 1 (HSV-1) and the episomal retention properties of the scaffold/matrix attachment region (S/MAR). The iBAC-S/MAR vector was used to deliver and maintain a 135 kb genomic DNA insert carrying the human low density lipoprotein receptor (LDLR) genomic DNA locus at high efficiency in CHO ldlr(-/-) a7 cells. Long-term studies on CHO ldlr(-/-) a7 clonal cell lines carrying iBAC-S/MAR-LDLR demonstrated low copy episomal stability of the vector for >100 cell generations without selection. Expression studies demonstrated that iBAC-S/MAR-LDLR completely restored LDLR function in CHO ldlr(-/-) a7 cells to physiological levels and that this expression can be repressed by approximately 70% by high sterol levels, recapitulating the same feedback regulation seen at the endogenous LDLR locus. This vector overcomes the major problems of vector integration and unregulated transgene expression.
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Registered trials
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.