RNA, in the context of therapeutic applications, is treated using techniques like RNA therapy, specifically with antisense oligonucleotides (AONs) or antisense oligos (ASOs). This involves using short, synthetic RNA strands designed to target specific RNA sequences within the body.
Here's a breakdown:
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Antisense Oligonucleotides (ASOs): ASOs are short sequences of synthetic nucleotides (DNA or RNA) designed to bind to specific RNA molecules within a cell. This binding can have several effects:
- Blocking Translation: ASOs can prevent the ribosome from binding to mRNA, thereby inhibiting protein synthesis.
- RNA Degradation: ASOs can trigger the degradation of the target RNA molecule by recruiting cellular enzymes like RNase H.
- Modulating RNA Splicing: ASOs can alter how pre-mRNA is spliced, leading to different protein isoforms or preventing the production of a disease-causing protein.
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Delivery: ASOs are typically delivered directly into the body, often via injection. Research is ongoing to improve delivery methods to enhance uptake by target tissues and cells. Chemical modifications to the ASO itself can improve stability, reduce immune response, and enhance its ability to reach the target RNA.
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Specificity: ASOs are designed to be highly specific for their target RNA sequence, minimizing off-target effects. However, careful design and testing are crucial to ensure specificity.
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Example Application: RNA therapy is being investigated for a range of diseases, including genetic disorders (like Duchenne Muscular Dystrophy), cancers, and infectious diseases.
Therefore, "treating RNA" often means using synthetic RNA or DNA molecules to interact with existing RNA within cells to modify gene expression or RNA function, ultimately to treat disease.
