ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector).
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2013. 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, 34 citations in OpenAlex.
- Viral vector-based gene therapies in the clinic: An update.Bioengineering & translational medicine · 2026Review
- Cell-based assay to detect small molecules restoring levels of let-7 miRNAs.American journal of cancer research · 2024Article
- Interferon-inducible phospholipids govern IFITM3-dependent endosomal antiviral immunity.The EMBO journal · 2023Article
- Spatiotemporal quantitative microRNA-155 imaging reports immune-mediated changes in a triple-negative breast cancer model.Frontiers in immunology · 2023Article
- Live-cell imaging of microRNA expression with post-transcriptional feedback control.Molecular therapy. Nucleic acids · 2021Article
- LentiRILES, a miRNA-ON sensor system for monitoring the functionality of miRNA in cancer biology and therapy.RNA biology · 2021Article
- Deletion of a pseudogene within a fragile site triggers the oncogenic expression of the mitotic CCSER1 gene.Life science alliance · 2021Article
- Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.Blood advances · 2021Article
- Genetically Encoded Reporter Genes for MicroRNA Imaging in Living Cells and Animals.Molecular therapy. Nucleic acids · 2020Review
- Development of Novel Riboswitches for Synthetic Biology in the Green Alga Chlamydomonas.ACS synthetic biology · 2020Article
- Cell-specific CRISPR-Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins.Nucleic acids research · 2019Article
- Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells.Cell stem cell · 2018Article
- MicroRNA-Regulated Gene Delivery Systems for Research and Therapeutic Purposes.Molecules (Basel, Switzerland) · 2018Review
- Article
- Regulation of HSVtk gene by endogenous microRNA-122a in liver cell lines as suicide gene therapy.Gastroenterology and hepatology from bed to bench · 2017Article
- Article
- MicroRNA-regulated viral vectors for gene therapy.World journal of experimental medicine · 2016Review
- Progresses towards safe and efficient gene therapy vectors.Oncotarget · 2015Review
- Rationally Designed MicroRNA-Based Genetic Classifiers Target Specific Neurons in the Brain.ACS synthetic biology · 2015Article
- Monitoring the spatiotemporal activities of miRNAs in small animal models using molecular imaging modalities.International journal of molecular sciences · 2015Review
Corrections and comments
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Authors and funding
4 authors at 3 institutions in 2 countries.
Funding
Abstract
To better understand and exploit microRNA (miR) regulation, a more precise characterization of miR expression patterns within a tissue or a lineage during development, differentiation, and homeostasis is needed. We previously showed that lentiviral vectors (LV) can be made responsive to miR to stringently control transgene expression as well as to report miR activity "live" and at the single-cell level. Although very useful, this approach reports miR activity by transgene suppression, hampering the direct identification and selection of miR-expressing cells. Here, we describe a strategy to couple transgene expression to the activity of the miR of interest. To this aim, we generated LV encoding two in-series OFF switches: a transcriptional repressor tagged with miR target sequences and a reporter cassette under the control of the repressor. Reporter expression is ON only when the miR is active and represses translation of the transcriptional repressor. We successfully applied this design to different types of repressors, multiple gene encoding vectors and delivered the system either by two separate or a self-contained vector. We demonstrated its performance by live monitoring of two miRs in different stages of human primary hematopoietic stem/progenitor cell differentiation in vivo. Further applications of this approach include imaging of rare miR-expressing cells and positive regulation of a therapeutic or selector gene in target cells identified by the expression of selected miRs.
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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.