Application of Antisense Oligonucleotides as an Alternative Approach for Gene Expression Control and Functional Studies

Antisense oligonucleotides (ASOs) are short, synthetic DNA fragments that offer a powerful means of modulating gene expression. By leveraging endogenous regulatory pathways, ASOs enable precise control over gene activity at multiple levels, including genomic DNA, transcription, RNA processing, and translation. Their applications span basic research and translational science, ranging from the generation of epigenetically modified organisms as potential GMO alternatives to the development of therapies for rare or treatment-resistant diseases. This review highlights the molecular mechanisms of ASO action, design and modification strategies, and delivery approaches across diverse cell types. Future directions include elucidating detailed molecular pathways, optimizing experimental conditions, and enhancing the persistence of therapeutic effects. Overall, ASOs represent a versatile and innovative tool in functional genomics, with broad implications for molecular biology, biotechnology, and medicine.