RNA Biomarkers and Drugs

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Biopharmaceuticals".

Deadline for manuscript submissions: closed (20 July 2023) | Viewed by 1365

Special Issue Editor

Department of Neurology, University of California at Davis, Sacramento, CA 95816, USA
Interests: microRNA; cardiomyopathy; microRNA therapeutics; hematoma; collagenases; brain ischemia; microglia

Special Issue Information

Dear Colleagues,

The RNA-based genomic biomarkers (e.g., gene (mRNA), non-coding RNA (ncRNA)), have been advancing our knowledge about the pathogenesis of individual diseases, such as cancers, neurological disorders, mental illnesses, and others. Since most human diseases are heterogenous and involve a large set of altered RNAs (e.g., mRNAs, ncRNAs) varying case by case, it is undoable to fully utilize genomic signatures for patient-tailored therapeutics, because most FDA-approved drugs (including small molecules, and RNA drugs, such as antisense oligonucleotides (ASO), small interfering RNAs (siRNA)) were developed on the basis of “one to one ligand-receptor theory”, which focuses on small molecules, with each molecule targeting a single protein or RNA. It appears that the high-throughput genomic data outgrow the “receptor theory”—the basic framework of drug development for more than a century.

By contrast, microRNA (miRNA, one of the most widely studied ncRNA) provides a new “one to hundreds drug-target” binding style, that is, a single miRNA can bind to and decrease hundreds of target genes, producing more robust efficacy than inhibiting a single gene. However, chemically modified miRNA drug development is still in its infancy, although dozens of miRNA drugs have entered clinical trials over the past decade.

Aside from RNA biomarkers and FDA-approved or ongoing trial RNA drugs (e.g., ASO, siRNA, miRNA), topics of other ncRNAs (e.g., long no-coding (lncRNA), circular RNAs (circRNAs), piwi-interacting RNAs (piRNAs)), guide RNAs (gRNA), RNA drug synthesis, RNA drug formulation and in vivo delivery, RNA drug side effects, and RNA-targeting small molecules are all suitable for this Special Issue.

Dr. Da Zhi Liu
Guest Editor

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Published Papers (1 paper)

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Review

22 pages, 1620 KiB  
Review
Tumor Suppressor MicroRNAs in Clinical and Preclinical Trials for Neurological Disorders
by Austin Lui, Timothy Do, Omar Alzayat, Nina Yu, Su Phyu, Hillary Joy Santuya, Benjamin Liang, Vidur Kailash, Dewey Liu, Sabra S. Inslicht, Kiarash Shahlaie and DaZhi Liu
Pharmaceuticals 2024, 17(4), 426; https://doi.org/10.3390/ph17040426 - 27 Mar 2024
Viewed by 965
Abstract
Cancers and neurological disorders are two major types of diseases in humans. We developed the concept called the “Aberrant Cell Cycle Disease (ACCD)” due to the accumulating evidence that shows that two different diseases share the common mechanism of aberrant cell cycle re-entry. [...] Read more.
Cancers and neurological disorders are two major types of diseases in humans. We developed the concept called the “Aberrant Cell Cycle Disease (ACCD)” due to the accumulating evidence that shows that two different diseases share the common mechanism of aberrant cell cycle re-entry. The aberrant cell cycle re-entry is manifested as kinase/oncoprotein activation and tumor suppressor (TS) inactivation, which are associated with both tumor growth in cancers and neuronal death in neurological disorders. Therefore, some cancer therapies (e.g., kinase/oncogene inhibition and TS elevation) can be leveraged for neurological treatments. MicroRNA (miR/miRNA) provides a new style of drug-target binding. For example, a single tumor suppressor miRNA (TS-miR/miRNA) can bind to and decrease tens of target kinases/oncogenes, producing much more robust efficacy to block cell cycle re-entry than inhibiting a single kinase/oncogene. In this review, we summarize the miRNAs that are altered in both cancers and neurological disorders, with an emphasis on miRNA drugs that have entered into clinical trials for neurological treatment. Full article
(This article belongs to the Special Issue RNA Biomarkers and Drugs)
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