Current Advances in RNA Therapeutics for Human Diseases

Abstract

Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were recognized as the genetic code containing the necessary information for proper cell functioning. In the years following these discoveries, vast knowledge of the seemingly endless roles of RNA have become better understood. Additionally, many new types of RNAs were discovered that seemed to have no coding properties (non-coding RNAs), such as microRNAs (miRNAs). The discovery of these new RNAs created a new avenue for treating various human diseases. However, RNA is relatively unstable and is degraded fairly rapidly once administered; this has led to the development of novel delivery mechanisms, such as nanoparticles to increase stability as well as to prevent off-target effects of these molecules. Current advances in RNA-based therapies have substantial promise in treating and preventing many human diseases and disorders through fixing the pathology instead of merely treating the symptomology similarly to traditional therapeutics. Although many RNA therapeutics have made it to clinical trials, only a few have been FDA approved thus far. Additionally, the results of clinical trials for RNA therapeutics have been ambivalent to date, with some studies demonstrating potent efficacy, whereas others have limited effectiveness and/or toxicity. Momentum is building in the clinic for RNA therapeutics; future clinical care of human diseases will likely comprise promising RNA therapeutics. This review focuses on the current advances of RNA therapeutics and addresses current challenges with their development.

microRNA-145-5p inhibits prostate cancer bone metastatic by modulating the epithelial-mesenchymal transition

“…However, RNA-based therapeutic drug delivery remain critical hurdles. Lipid-based nanoparticle delivery systems, containing a variety of effective RNA-base therapeutic payloads, can ensure specific delivery of therapeutic drugs to tissues and exert attractive properties (31)(32)(33). From the part of the clinical trials, RNA-based therapeutics will provide a promising treatment approach for various human diseases.…”

Targeting Fibronectin to Overcome Remyelination Failure in Multiple Sclerosis: The Need for Brain- and Lesion-Targeted Drug Delivery

“…By doing so, the need for frequent dosing is reduced. Nanovehicles can improve the bio-availability of hydrophobic and hydrophilic compounds by providing protection against chemical and biological degradation and improving target-site delivery [ 241 , 242 , 243 ]. Additionally, nanoparticle design can be optimized for the development of precision medicine, where the personal characteristics of patients in conjunction with specified nanomedicine engineering allow for patient-specific disease treatments [ 244 ].…”

MicroRNA-34a: A Novel Therapeutic Target in Fibrosis

“…Currently there are no FDA-approved miRNAs, but many miRNA therapies have achieved substantial preclinical efficacy, even entered in clinical trials ( Wang et al, 2021 ; Smith et al, 2022 ; Zogg et al, 2022 ). For example, miravirsen (miR-122 inhibitor) has completed Phase II clinical trials for the treatment of Hepatitis C ( Janssen et al, 2013 ; Panigrahi et al, 2022 ).…”