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Antisense Oligonucleotides: A Novel Therapeutic Approach for Genetic Diseases What are Antisense Oligonucleotides? Antisense oligonucleotides (ASOs) are short, synthetic, chemically modified strands of nucleic acids that are used to modulate gene expression by binding to target messenger RNA (mRNA) through complementary base pairing. The binding inhibits or modulates the expression of specific genes, thereby regulating the production of disease-causing proteins. ASOs were first proposed as a therapeutic approach in the late 1970s but have advanced significantly in recent years due to improved chemistries, delivery methods, and understanding of RNA biology. Mechanism of Action ASOs work by binding to their target mRNA through Watson-Crick base pairing. Depending on the type of antisense oligonucleotides used, this binding can then activate or inhibit critical steps in gene expression. Most commonly used ASOs are designed to induce RNase H-mediated degradation of the mRNA target. RNase H is an endogenous endonuclease that is activated upon binding of an ASO/RNA duplex and cleaves the RNA strand, leading to reduced levels of the target mRNA and consequently lower production of the encoded protein. Other ASO mechanisms involve steric hindrance blocking of pre-mRNA splicing or translational machinery, leading to exon skipping or inhibiting translation respectively. Well-designed ASOs can selectively regulate expression of one gene among thousands to obtain a therapeutic effect. Get More Insights on Antisense oligonucleotides https://fortunetelleroracle.com/health-fitness/antisense-oligonucleotides--a-novel-therapeutic-approach-for-genetic-diseases-962536