ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Conditional Regulation of Gene Expression by Ligand-Induced Occlusion of a MicroRNA Target Sequence.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. 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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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
22 citing papers in PubMed, 29 citations in OpenAlex.
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- Engineering aptamer dimers (apdimers) for optimization of synthetic riboswitches.Nucleic acids research · 2026Article
- Enhancing small-molecule-mediated translational control through multivalent RNA aptamers.Nucleic acids research · 2026Article
- Engineering high-titer lentiviral vectors for robust expression of RNA-based gene circuits.bioRxiv : the preprint server for biology · 2026Article
- Potential and Optimization of Mammalian Splice Riboswitches for the Regulation of Exon Skipping-Dependent Gene Expression and Isoform Switching within theACS synthetic biology · 2025Article
- Engineering oxypurinol-responsive riboswitches based on bacterial xanthine aptamers for gene expression control in mammalian cell culture.Nucleic acids research · 2025Article
- Engineered Branaplam Aptamers Exploit Structural Elements from Natural Riboswitches.ACS chemical biology · 2024Article
- RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes.Nature communications · 2023Article
- Exploiting natural riboswitches for aptamer engineering and validation.Nucleic acids research · 2023Article
- Tapping the potential of synthetic riboswitches: reviewing the versatility of the tetracycline aptamer.RNA biology · 2023Review
- Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver.PloS one · 2023Article
- Next RNA Therapeutics: The Mine of Non-Coding.International journal of molecular sciences · 2022Review
- Efficient splicing-based RNA regulators for tetracycline-inducible gene expression in human cell culture and C. elegans.Nucleic acids research · 2021Article
- Riboswitches for Controlled Expression of Therapeutic Transgenes Delivered by Adeno-Associated Viral Vectors.Pharmaceuticals (Basel, Switzerland) · 2021Review
- Translational control of enzyme scavenger expression with toxin-induced micro RNA switches.Scientific reports · 2021Article
- AAV Vectored Immunoprophylaxis for Filovirus Infections.Tropical medicine and infectious disease · 2020Review
- Selection of High-Affinity RNA Aptamers That Distinguish between Doxycycline and Tetracycline.Biochemistry · 2020Article
- High-throughput identification of synthetic riboswitches by barcode-free amplicon-sequencing in human cells.Nature communications · 2020Article
- Promise and Progress of an HIV-1 Cure by Adeno-Associated Virus Vector Delivery of Anti-HIV-1 Biologics.Frontiers in cellular and infection microbiology · 2020Review
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Authors and funding
7 authors at 1 institution in 2 countries.
Funding
Abstract
RNA switches that modulate gene expression with small molecules have a number of scientific and clinical applications. Here, we describe a novel class of small regulatory on switches based on the ability of a ligand-bound aptamer to promote stem formation between a microRNA target sequence (miR-T) and a complementary competing strand. Two on switch architectures employing this basic concept were evaluated, differing in the location of a tetracycline aptamer and the region of a miR-21 target sequence (miR-21-T) masked by its competing strand. Further optimizations of miR-21-T and its competing strand resulted in tetracycline-regulated on switches that induced luciferase expression by 19-fold in HeLa cells. A similar switch design based on miR-122-T afforded 7-fold regulation when placed in tandem, indicating that this approach can be extended to additional miR-T. Optimized on switches introduced into adeno-associated virus (AAV) vectors afforded 10-fold regulation of two antiviral proteins in AAV-transduced cells. Our data demonstrate that small-molecule-induced occlusion of a miR-T can be used to conditionally regulate gene expression in mammalian cells and suggest that regulatory switches built on this principle can be used to dose expression of an AAV transgene.
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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.