ArticleMolecular therapy. Nucleic acids2014
Designing Ago2-specific siRNA/shRNA to Avoid Competition with Endogenous miRNAs.
Article in Molecular therapy. Nucleic acids, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
34 citing papers in PubMed, 43 citations in OpenAlex.
- Understanding the biological limits of hybridization-dependent siRNA off-target interactions.Molecular therapy. Nucleic acids · 2026Article
- Small RNA or oligonucleotide drugs and challenges in evaluating drug-drug interactions.Frontiers in pharmacology · 2025Review
- siRNA Silencing of Adipogenic Gene in ASCs.Methods in molecular biology (Clifton, N.J.) · 2025Article
- LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders.Molecular therapy. Nucleic acids · 2024Article
- Subretinal AAV delivery of RNAi-therapeutics targetingMolecular therapy. Methods & clinical development · 2024Article
- miRNA Pathway Alteration in Response to Non-Coding RNA Delivery in Viral Vector-Based Gene Therapy.International journal of molecular sciences · 2022Review
- Article
- miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.Nucleic acid therapeutics · 2022Article
- Non-targeting control for MISSION shRNA library silencesMolecular therapy. Nucleic acids · 2021Article
- MicroRNAs Regulating Autophagy in Neurodegeneration.Advances in experimental medicine and biology · 2021Article
- Short Hairpin RNAs for Strand-Specific Small Interfering RNA Production.Frontiers in bioengineering and biotechnology · 2020Article
- Article
- Differences in silencing of mismatched targets by sliced versus diced siRNAs.Nucleic acids research · 2018Article
- Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Review
- Dicer-independent processing of small RNA duplexes: mechanistic insights and applications.Nucleic acids research · 2017Review
- AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.Gene therapy · 2017Article
- A Simple and Cost-Effective Approach for In Vitro Production of Sliced siRNAs as Potent Triggers for RNAi.Molecular therapy. Nucleic acids · 2017Article
- Silencing of HIV-1 by AgoshRNA molecules.Gene therapy · 2017Article
- Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.Molecular therapy. Methods & clinical development · 2017Article
- The influence of the 5΄-terminal nucleotide on AgoshRNA activity and biogenesis: importance of the polymerase III transcription initiation site.Nucleic acids research · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Relatively large amounts of transfected siRNA can compete for Ago proteins and thus compromise endogenous miRNA function, potentially leading to toxicities. Here, we show that shRNA can also perturb endogenous miRNA function similarly. More importantly, we also show that the problem can be solved by designing shRNAs in the context of pre-miR-451 structure with completely complementary stem, which significantly improves the Ago2 specificity. This shRNA was shown to be Ago2-specific, and maintain target-silencing ability while avoiding competition with endogenous miRNAs by not competing for Agos 1, 3, and 4. We conclude that modified pre-miR-451 structure provides a general platform to design shRNAs that significantly reduce perturbation of miRNA function.
Identifiers
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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.