Targeting RNA Coding Mechanisms in Disease Molecular Pathways

A special issue of Genes (ISSN 2073-4425). This special issue belongs to the section "RNA".

Deadline for manuscript submissions: 20 January 2027 | Viewed by 612

Editor


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Guest Editor
Department of Chemistry, University of Tennessee at Chattanooga, Chattanooga, TN 37403, USA
Interests: nucleic acid-targeted small molecules; chemical gene modification

Special Issue Information

Dear Colleagues,

In recent years, RNA has become a key player in the biology of diseases, evolving beyond its traditional role as a mere messenger to uncover complex and dynamic regulatory functions. Disruptions in RNA coding mechanisms—such as alternative splicing, the function of non-coding RNA (ncRNA) and RNA modifications—are increasingly recognized as significant contributors to the development of various diseases, including cancer, cardiovascular disorders and neurological conditions.

This Special Issue titled "Targeting RNA Coding Mechanisms in Disease Molecular Pathways" explores the latest advances in understanding how RNA dysregulation contributes to these diseases and highlights innovative therapeutic strategies. We invite submissions that detail novel RNA-focused drug discovery approaches, examine the functional roles of ncRNAs and epitranscriptomics in disease pathways and introduce new technologies for manipulating RNA coding mechanisms. By concentrating on this transformative area of research, we aim to provide a comprehensive overview of the field and stimulate the development of next-generation diagnostics and therapies targeting RNA.

Dr. Wangyong Yang
Guest Editor

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Keywords

  • RNA therapeutics
  • RNA modifications (epitranscriptomics)
  • non-coding RNA (ncRNA)
  • alternative splicing
  • RNA-binding proteins (RBPs)
  • RNA-targeted small molecules
  • gene therapy
  • molecular pathways
  • disease biomarkers
  • RNA vaccines

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Published Papers (1 paper)

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Review

29 pages, 2229 KB  
Review
Beyond Coding Variants: RNA-Level Mechanisms in Human Disease and Precision Therapeutics
by Himanshu Goel
Genes 2026, 17(7), 777; https://doi.org/10.3390/genes17070777 - 30 Jun 2026
Viewed by 151
Abstract
Clinical genomics has traditionally focused on protein-coding variation, yet many pathogenic mechanisms arise through alterations in RNA processing, stability, localisation, translation, and surveillance. Prior reviews have addressed individual RNA layers, splicing, non-coding RNAs, RNA therapeutics, or RNA diagnostics in isolation. This review presents [...] Read more.
Clinical genomics has traditionally focused on protein-coding variation, yet many pathogenic mechanisms arise through alterations in RNA processing, stability, localisation, translation, and surveillance. Prior reviews have addressed individual RNA layers, splicing, non-coding RNAs, RNA therapeutics, or RNA diagnostics in isolation. This review presents an integrated, mechanism-matched framework linking RNA-level disease mechanisms to diagnostic reasoning and therapeutic selection across all major RNA layers, offering a practical resource for clinical geneticists and translational researchers. I examine how splicing defects, pseudoexon inclusion, polyadenylation disruption, RNA editing loss, untranslated-region variants, premature termination codons, stop-loss variants, RNA-binding protein dysfunction, non-coding RNA dysregulation, altered codon usage, ribosome stalling, and surveillance pathway failure, including nonsense-mediated decay, nonstop decay, and no-go decay, each create distinct and mechanistically addressable disease states. A central argument of this review is that treatment selection must be mechanism-matched rather than gene- or variant-class-based: splice defects may require antisense oligonucleotide (ASO)-mediated correction or small-molecule splice modulation; toxic transcripts may require ASO- or siRNA-mediated silencing; haploinsufficiency may require mRNA replacement or transcript rescue; premature termination codons are candidates for readthrough only when transcript and protein context are favourable. I further argue that RNA sequencing, long-read transcriptomics, allele-specific expression analysis, and functional assays are essential for both diagnosis and therapeutic stratification. The framework described here moves clinical variant interpretation beyond descriptive classification toward mechanism-based, RNA-centric precision medicine. Full article
(This article belongs to the Special Issue Targeting RNA Coding Mechanisms in Disease Molecular Pathways)
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