Non-coding RNAs (ncRNAs) account for approximately 99% of the human transcribed genome, with the number of validated ncRNAs growing every year since the cost of next-generation sequencing technologies has greatly declined over the past two decades. A paradigm shift has occurred in our understanding of ncRNA as a functionally important class of molecules. Unlike messenger RNA (mRNA), these sequences lack the potential to encode proteins or peptides, but rather serve as controllers in the epigenetic, post-transcriptional, and translational regulation of gene expression. Interestingly, a large number of ncRNAs, such as microRNAs (miRNAs), circular RNAs (circRNAs), small nuclear RNAs (snRNAs), piwi-interacting RNAs (piRNAs) and long non-coding RNAs (lncRNAs), have increasingly been reported to engage in various genetic disorders, which lead to numerous complex and intractable pathologies.
Many illnesses are now considered “diseases of the genome,” carrying a genetic component or being triggered by a genetic predisposition. According to the OMIM database updated in November 2022, there are over 7000 known genetic disorders, including monogenic, polygenic, and chromosomal abnormalities. Realizing that ncRNA is the major product of the genome therefore immediately raises the question of how ncRNAs contribute to the progression of genetic diseases. Meanwhile, overwhelming evidence indicate that ncRNAs appear to constitute a hidden layer of molecular genetic signals. Although most mutations in these regulatory sequences may be either subtle or difficult to detect, they can nonetheless cause profoundly debilitating and diverse pathologies. Moreover, it is becoming evident that ncRNAs, which are highly expressed in a variety of tissues, not only serve as the primary orchestrators of crucial gene regulatory networks regulating various cellular processes but also have versatile modes of action, indicating that they can be potential therapeutic targets for genetic disorders. However, the process of discovering therapeutic targets is never easy, particularly when it comes to developing drugs to target ncRNAs. The rapid evolution of ncRNAs and the uncertainty about their functions and mechanisms of action make lead identification and development even more challenging.
Regarding this Research Topic, we welcome scientists to submit Original Research, Clinical Trials, Case Reports, Reviews, Systematic Reviews and Mini Reviews covering but not limited to the following themes:
• Exploration of the therapeutic potential of ncRNAs in genetic disorders
• Post-transcriptional, translational, and post-translational regulation of gene expression by targeting ncRNAs in genetic disorders
• Emerging role of extracellular ncRNAs in genetic disorders and their potential for precision medicine
• Identification of novel ncRNA-based targets from the mitochondrial genome, nuclear genome, and microbial metagenome
• Delivery of ncRNA-based therapeutics for treating genetic disorders
• Elucidation of the ncRNA-based regulatory system using single-cell multi-omics and its application for drug discovery
Please note: If patient data are analyzed (datasets included), a comprehensive description of the patients including sex, age, diagnostic criteria, inclusion and exclusion criteria, disease stage, therapy received, comorbidities as well as additional clinical information and assessment of clinical response/effects should be included. If genetic, proteomics, metabolomics, or other omics data are analyzed, a comprehensive description of the methods and the rationale for the selection of the specific data studied should be provided. Studies related to natural compounds, herbal extracts, or traditional medicine products, are outside the scope of this Research Topic and should instead be submitted to the specialty section Ethnopharmacology.
Non-coding RNAs (ncRNAs) account for approximately 99% of the human transcribed genome, with the number of validated ncRNAs growing every year since the cost of next-generation sequencing technologies has greatly declined over the past two decades. A paradigm shift has occurred in our understanding of ncRNA as a functionally important class of molecules. Unlike messenger RNA (mRNA), these sequences lack the potential to encode proteins or peptides, but rather serve as controllers in the epigenetic, post-transcriptional, and translational regulation of gene expression. Interestingly, a large number of ncRNAs, such as microRNAs (miRNAs), circular RNAs (circRNAs), small nuclear RNAs (snRNAs), piwi-interacting RNAs (piRNAs) and long non-coding RNAs (lncRNAs), have increasingly been reported to engage in various genetic disorders, which lead to numerous complex and intractable pathologies.
Many illnesses are now considered “diseases of the genome,” carrying a genetic component or being triggered by a genetic predisposition. According to the OMIM database updated in November 2022, there are over 7000 known genetic disorders, including monogenic, polygenic, and chromosomal abnormalities. Realizing that ncRNA is the major product of the genome therefore immediately raises the question of how ncRNAs contribute to the progression of genetic diseases. Meanwhile, overwhelming evidence indicate that ncRNAs appear to constitute a hidden layer of molecular genetic signals. Although most mutations in these regulatory sequences may be either subtle or difficult to detect, they can nonetheless cause profoundly debilitating and diverse pathologies. Moreover, it is becoming evident that ncRNAs, which are highly expressed in a variety of tissues, not only serve as the primary orchestrators of crucial gene regulatory networks regulating various cellular processes but also have versatile modes of action, indicating that they can be potential therapeutic targets for genetic disorders. However, the process of discovering therapeutic targets is never easy, particularly when it comes to developing drugs to target ncRNAs. The rapid evolution of ncRNAs and the uncertainty about their functions and mechanisms of action make lead identification and development even more challenging.
Regarding this Research Topic, we welcome scientists to submit Original Research, Clinical Trials, Case Reports, Reviews, Systematic Reviews and Mini Reviews covering but not limited to the following themes:
• Exploration of the therapeutic potential of ncRNAs in genetic disorders
• Post-transcriptional, translational, and post-translational regulation of gene expression by targeting ncRNAs in genetic disorders
• Emerging role of extracellular ncRNAs in genetic disorders and their potential for precision medicine
• Identification of novel ncRNA-based targets from the mitochondrial genome, nuclear genome, and microbial metagenome
• Delivery of ncRNA-based therapeutics for treating genetic disorders
• Elucidation of the ncRNA-based regulatory system using single-cell multi-omics and its application for drug discovery
Please note: If patient data are analyzed (datasets included), a comprehensive description of the patients including sex, age, diagnostic criteria, inclusion and exclusion criteria, disease stage, therapy received, comorbidities as well as additional clinical information and assessment of clinical response/effects should be included. If genetic, proteomics, metabolomics, or other omics data are analyzed, a comprehensive description of the methods and the rationale for the selection of the specific data studied should be provided. Studies related to natural compounds, herbal extracts, or traditional medicine products, are outside the scope of this Research Topic and should instead be submitted to the specialty section Ethnopharmacology.
