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Antisense oligonucleotides (ASOs) are short, synthetic strands of nucleic acids designed to bind to specific mRNA sequences. Their purpose is to modify gene expression by blocking, altering, or enhancing protein production at the RNA level. ASOs have gained attention as versatile therapeutic agents due to their capacity for high specificity.

These molecules operate by complementary base pairing, a principle fundamental to genetics. When an ASO binds to its target mRNA, it can trigger different outcomes. Some ASOs promote degradation of the mRNA through cellular enzymes, effectively reducing the production of harmful proteins. Others alter splicing, allowing cells to produce functional versions of proteins that are otherwise defective in genetic diseases.

One of the most notable strengths of ASOs is their adaptability. As long as the genetic sequence of interest is known, an ASO can be designed to target it. This makes ASOs promising for rare diseases, many of which arise…