ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection.
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 17 papers.
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Who cites it
17 citing papers in PubMed, 25 citations in OpenAlex.
- Therapeutic Applications of Poly-miRNAs and miRNA Sponges.International journal of molecular sciences · 2025Review
- Oncogenic Viruses-Encoded microRNAs and Their Role in the Progression of Cancer: Emerging Targets for Antiviral and Anticancer Therapies.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Recombinant adeno-associated virus serotype 9 AAV-RABVG expressing a Rabies Virus G protein confers long-lasting immune responses in mice and non-human primates.Emerging microbes & infections · 2022Article
- Engineering, delivery, and biological validation of artificial microRNA clusters for gene therapy applications.Nature protocols · 2019Article
- Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy.Journal of visualized experiments : JoVE · 2019Article
- Optimizing Synthetic miRNA Minigene Architecture for Efficient miRNA Hairpin Concatenation and Multi-target Gene Knockdown.Molecular therapy. Nucleic acids · 2019Article
- Enterovirus 71 Suppresses miR-17-92 Cluster Through Up-Regulating Methylation of the miRNA Promoter.Frontiers in microbiology · 2019Article
- miRNAs regulate immune response and signaling during hepatitis C virus infection.European journal of medical research · 2018Review
- Improving miRNA Delivery by Optimizing miRNA Expression Cassettes in Diverse Virus Vectors.Human gene therapy methods · 2017Review
- TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA locus.Nucleic acids research · 2017Article
- Developing miRNA therapeutics for cardiac repair in ischemic heart disease.Journal of thoracic disease · 2016Review
- MicroRNAs and Cardiac Regeneration.Circulation research · 2015Review
- Baculovirus-mediated miRNA regulation to suppress hepatocellular carcinoma tumorigenicity and metastasis.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- MicroRNA and hepatitis C virus--challenges in investigation and translation: a review of the literature.Diagnostic microbiology and infectious disease · 2014Review
- MicroRNAs as regulators of cutaneous wound healing.Journal of biosciences · 2014Review
- Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Review
- Hepatitis C virus infection, microRNA and liver disease progression.World journal of hepatology · 2013Review
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We developed a strategy to treat hepatitis C virus (HCV) infection by replacing five endogenous microRNA (miRNA) sequences of a natural miRNA cluster (miR-17-92) with sequences that are complementary to the HCV genome. This miRNA cluster (HCV-miR-Cluster 5) is delivered to cells using adeno-associated virus (AAV) vectors and the miRNAs are expressed in the liver, the site of HCV replication and assembly. AAV-HCV-miR-Cluster 5 inhibited bona fide HCV replication in vitro by up to 95% within 2 days, and the spread of HCV to uninfected cells was prevented by continuous expression of the anti-HCV miRNAs. Furthermore, the number of cells harboring HCV RNA replicons decreased dramatically by sustained expression of the anti-HCV miRNAs, suggesting that the vector is capable of curing cells of HCV. Delivery of AAV-HCV-miR-Cluster 5 to mice resulted in efficient transfer of the miRNA gene cluster and expression of all five miRNAs in liver tissue, at levels up to 1,300 copies/cell. These levels achieved up to 98% gene silencing of cognate HCV sequences, and no liver toxicity was observed, supporting the safety of this approach. Therefore, AAV-HCV-miR-Cluster 5 represents a different paradigm for the treatment of HCV infection.
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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.