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Disease-related messenger RNA (mRNA (contextually: disease-related mRNA))
- Target
- mRNA (contextually: disease-related mRNA)
- Molecular classification
- Other (mRNA is a nucleic acid, not a receptor, enzyme, transporter, etc.), RNA molecule, Coding RNA
01
Overview
Disease-related messenger RNA is any mRNA molecule that is directly implicated in human disease, either through abnormal expression, splicing, sequence, or function. Aberrant mRNA levels and processing can drive pathogenic mechanisms in cancer, genetic disorders, inflammation, neurodegenerative diseases, and infections. Therapeutic approaches targeting disease-related mRNAs include gene editing, RNA interference, antisense oligonucleotides, and mRNA-based vaccines, which prevent or correct disease by modulating protein expression at the mRNA level[2][6][9][10]. Disease-related mRNA also serves as a highly informative biomarker for diagnosis, prognosis, and patient stratification in modern precision medicine[5][9][10].
- Other names
- Pathogenic mRNAAberrant mRNADisease-associated mRNADysfunctional mRNA
02
Mechanism of action
Translation inhibition (by ASOs or siRNA); mRNA degradation (RNAi); mRNA replacement (encode therapeutic/functional protein); mRNA-based vaccination (induce immune response to specific antigens)
03
Biological functions
Protein synthesis (serves as template for translation)Gene expression regulation (expression level and splicing changes)Cell signaling (in some settings)Biomarker (for diagnosis, prognosis)
04
Disease associations
Cancer (e.g. oncogene mRNA overexpression)Neurodegenerative disease (e.g. Alzheimer's, Parkinson's disease mRNAs)Cardiovascular disease (specific mRNA patterns)Genetic disorders (cystic fibrosis, ataxia, others)Inflammation (e.g. autoimmune disease-related mRNAs)Infection (e.g. response mRNA signatures in viral diseases)
05
Safety considerations
Off-target effects (siRNA/ASO may affect unintended mRNAs)Immune activation/inflammatory responsesStability and delivery challenges for therapeutic mRNARisk of toxicity due to overexpression or inappropriate protein synthesisDifficulties in specific targeting within tissues
06
Interacting drugs
mRNA vaccines (COVID-19 mRNA vaccine)
5 more in the full profile.
07
Biomarkers
mRNA expression signatures for disease diagnosis and prognosis (e.g. specific oncogene mRNAs in cancer, inflammatory mRNA markers in autoimmune disease)mRNA levels of disease-relevant genes for patient selection and monitoring
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