DNA, RNA, and antisense oligonucleotide (ASO) therapeutics work by targeting genetic material at the molecular level to regulate gene expression and treat diseases. DNA-based therapies, such as gene therapy, introduce or modify DNA sequences within cells to correct genetic defects or produce therapeutic proteins. RNA therapeutics, including mRNA vaccines and small interfering RNA (siRNA), work by either encoding proteins (mRNA) or silencing specific genes (siRNA, microRNA) to prevent disease-causing protein production. ASO therapeutics are short, synthetic DNA or RNA strands that bind to specific mRNA molecules, blocking translation or triggering degradation to reduce harmful protein levels. These approaches offer highly specific treatments for genetic disorders, cancers, and viral infections.
https://www.biotechnologyreviews.com/p/nucleic-acid-therapeutics-asos-sirna?r=45cg9n
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https://www.biotechnologyreviews.com/p/genetic-medicine-understanding-antisense?r=45cg9n
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