Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2022 Jul 5;23(13):7471.
doi: 10.3390/ijms23137471.

Next RNA Therapeutics: The Mine of Non-Coding

Sabrina Garbo  1 , Rossella Maione  1 , Marco Tripodi  1   2 , Cecilia Battistelli  1
Affiliations
Review

Next RNA Therapeutics: The Mine of Non-Coding

Sabrina Garbo et al. Int J Mol Sci. .

Abstract

The growing knowledge on several classes of non-coding RNAs (ncRNAs) and their different functional roles has aroused great interest in the scientific community. Beyond the Central Dogma of Biology, it is clearly known that not all RNAs code for protein products, and they exert a broader repertoire of biological functions. As described in this review, ncRNAs participate in gene expression regulation both at transcriptional and post-transcriptional levels and represent critical elements driving and controlling pathophysiological processes in multicellular organisms. For this reason, in recent years, a great boost was given to ncRNA-based strategies with potential therapeutic abilities, and nowadays, the use of RNA molecules is experimentally validated and actually exploited in clinics to counteract several diseases. In this review, we summarize the principal classes of therapeutic ncRNA molecules that are potentially implied in disease onset and progression, which are already used in clinics or under clinical trials, highlighting the advantages and the need for a targeted therapeutic strategy design. Furthermore, we discuss the benefits and the limits of RNA therapeutics and the ongoing development of delivery strategies to limit the off-target effects and to increase the translational application.

Keywords: RNA therapeutics; delivery strategies; ncRNAs.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflict of interest. The funders had no role in the management of the manuscript.

Figures

Figure 1
Figure 1
Schematic representation of hammerhead ribozymes mechanism of action.
Figure 2
Figure 2
Schematic representation of (A) CIRTS, (B) RESCUE, (C) RESTORE, and (D) LEAPER RNA editing approaches.
Figure 2
Figure 2
Schematic representation of (A) CIRTS, (B) RESCUE, (C) RESTORE, and (D) LEAPER RNA editing approaches.
Figure 3
Figure 3
Schematic representation of aptamers engineering on receptor inhibition, drug delivery, and therapeutic RNA vehiculation.
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Gao Y., Patil S., Qian A. The Role of MicroRNAs in Bone Metabolism and Disease. Int. J. Mol. Sci. 2020;21:6081. doi: 10.3390/ijms21176081. - DOI - PMC - PubMed
    1. Ali W., Spengler G., Kincses A., Nove M., Battistelli C., Latacz G., Starek M., Dabrowska M., Honkisz-Orzechowska E., Romanelli A., et al. Discovery of phenylselenoether-hydantoin hybrids as ABCB1 efflux pump modulating agents with cytotoxic and antiproliferative actions in resistant T-lymphoma. Eur. J. Med. Chem. 2020;200:112435. doi: 10.1016/j.ejmech.2020.112435. - DOI - PubMed
    1. Magistri P., Battistelli C., Strippoli R., Petrucciani N., Pellinen T., Rossi L., Mangogna L., Aurello P., D’Angelo F., Tripodi M., et al. SMO Inhibition Modulates Cellular Plasticity and Invasiveness in Colorectal Cancer. Front. Pharmacol. 2017;8:956. doi: 10.3389/fphar.2017.00956. - DOI - PMC - PubMed
    1. Rossi L., Battistelli C., de Turris V., Noce V., Zwergel C., Valente S., Moioli A., Manzione A., Palladino M., Bordoni V., et al. HDAC1 inhibition by MS-275 in mesothelial cells limits cellular invasion and promotes MMT reversal. Sci. Rep. 2018;8:8492. doi: 10.1038/s41598-018-26319-2. - DOI - PMC - PubMed
    1. Stazi G., Battistelli C., Piano V., Mazzone R., Marrocco B., Marchese S., Louie S.M., Zwergel C., Antonini L., Patsilinakos A., et al. Development of alkyl glycerone phosphate synthase inhibitors: Structure-activity relationship and effects on ether lipids and epithelial-mesenchymal transition in cancer cells. Eur. J. Med. Chem. 2019;163:722–735. doi: 10.1016/j.ejmech.2018.11.050. - DOI - PubMed

MeSH terms