Emerging RNA-based drugs: siRNAs, microRNAs and derivates
Abstract
An emerging new category of therapeutic agents based on ribonucleic acid has emerged and shown very promising in vitro, animal and pre-clinical results, known as small interfering RNAs (siRNAs), microRNAs mimics (miRNA mimics) and their derivates. siRNAs are small RNA molecules that promote potent and specific silencing of mutant, exogenous or aberrant genes through a mechanism known as RNA interference. These agents have called special attention to medicine since they have been used to experimentally treat a series of neurological conditions with distinct etiologies such as prion, viral, bacterial, fungal, genetic disorders and others. siRNAs have also been tested in other scenarios such as: control of anxiety, alcohol consumption, drug-receptor blockage and inhibition of pain signaling. Although in a much earlier stage, miRNAs mimics, anti-miRs and small activating RNAs (saRNAs) also promise novel therapeutic approaches to control gene expression. In this review we intend to introduce clinicians and medical researchers to the most recent advances in the world of siRNA- and miRNA-mediated gene control, its history, applications in cells, animals and humans, delivery methods (an yet unsolved hurdle), current status and possible applications in future clinical practice.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
A variety of RNA therapeutics have been developed for GARD therapies based on miRNA, siRNA, mRNA, lncRNA, and genome editing systems containing RNA components.
“To date, a variety of RNA therapeutics have been developed for GARD therapies based on the versatile family of RNA molecules, including microRNA (miRNA), small interfering RNA (siRNA), messenger RNA (mRNA), long non-coding RNA (lncRNA), and genome editing systems containing RNA components.[6, 9, 16]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [16]
Cited by
Sorted by citations
Long non-coding RNAs in cancer: implications for personalized therapy
Vitiello, M., Tuccoli, A., & Poliseno, L. (2014) · Cellular Oncology · doi:10.1007/s13402-014-0180-x · Cited by 107 · see citing works
RNA delivery biomaterials for the treatment of genetic and rare diseases
Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82 · see citing works
“[6, 9, 16] These different RNA-based therapeutics can be used to treat genetic diseases, primarily at the RNA level via distinct mechanisms of action.”
BackgroundComprehensive overview and assessment of computational prediction of microRNA targets in animals
Fan, X., & Kurgan, L. (2014) · Briefings in Bioinformatics · doi:10.1093/bib/bbu044 · Cited by 74 · see citing works
“MiRNAs are used to study signal transduction and pathogenesis of genetic [1–3], neurodegenerative [4] and metabolic diseases [5]”
BackgroundPitfalls in RNA Modification Quantification Using Nucleoside Mass Spectrometry
Ammann, G., Berg, M., Dalwigk, J. F., et al. (2023) · Accounts of Chemical Research · doi:10.1021/acs.accounts.3c00402 · Cited by 45 · see citing works
MiR-24 Is Required for Hematopoietic Differentiation of Mouse Embryonic Stem Cells
Roy, L., Bikorimana, E., Lapid, D., Choi, H., Nguyen, T. A., & Dahl, R. E. (2015) · PLoS Genetics · doi:10.1371/journal.pgen.1004959 · Cited by 25 · see citing works
Kitamura, Y., Masegi, Y., Ogawa, S., et al. (2013) · Bioorganic & Medicinal Chemistry · doi:10.1016/j.bmc.2013.05.046 · Cited by 13 · see citing works
Li, C., Liu, H., Zhou, Y., et al. (2020) · Chinese Medical Journal · doi:10.1097/cm9.0000000000001072 · Cited by 8 · see citing works
Therapeutic Implication of miRNAs as an Active Regulatory Player in the Management of Pain: A Review
Hassan, M., Shahzadi, S., Yasir, M., Chun, W., & Kloczkowski, A. (2024) · Genes · doi:10.3390/genes15081003 · Cited by 8 · see citing works
Methods and advances in RNA characterization and design
Contreras, L. M. (2018) · Methods · doi:10.1016/j.ymeth.2018.06.003 · Cited by 1 · see citing works
High-throughput Computational Characterization and Prediction of MicroRNA Targets
Fan, X. (2016) · ERA: Education and Research Archive (University of Alberta) · doi:10.7939/r3g15tn59
Brainin, M. (2013) · World Stroke Academy · doi:10.1002/wsa2.20020
Gene Therapy Strategies for Prophylactic and Therapeutic Treatments of Human Prion Diseases
Camacho, M. V., & Kong, Q. (2023) · Springer eBooks · doi:10.1007/978-3-031-20565-1_36
Hofseth, L. J., Weston, A., & Harris, C. C. (2017) · Holland‐Frei Cancer Medicine · doi:10.1002/9781119000822.hfcm023
- Authors
- Campos Pereira, T., & Lopes-Cendes, I.
- Venue
- Central Nervous System Agents in Medicinal Chemistry
- Publisher
- Bentham Science Publishers Ltd.
- Published
- 2012-08-01
- Volume
- 12
- Issue
- 3
- Pages
- 217-232
- ISSN
- 1568-0150 (ISSN-L)
- 1871-5249
- OpenAlex
- W1984498288
- Cited by
- 13
- References
- 0
- Citations by year
- 2024: 1 · 2023: 2 · 2020: 1 · 2019: 1 · 2018: 1 · 2017: 1 · 2016: 1 · 2015: 1 · 2014: 2 · 2013: 2
- FWCI
- 0.64
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Central Nervous System Agents in Medicinal Chemistry (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cancer Research › MicroRNA in disease regulation
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Keywords
- RNA therapeutics, siRNA, miRNA mimics, RNA interference, gene silencing, antimiRs, small activating RNA, RNA delivery, neurological disorders, prion disease, viral infection, genetic disorders, anxiety, alcohol consumption, pain signaling, gene expression regulation
- MeSH terms
- Animals, Mental Disorders, Pharmaceutical Preparations, Humans, Nervous System Diseases, Genetic Therapy, Gene Transfer Techniques, RNA, Small Interfering, MicroRNAs, Drug Discovery
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
