Genetic Medicine, Understanding Antisense Oligonucleotides ASOs, Design, modifications and in vivo
1Description
Antisense oligonucleotides (ASOs) represent a pivotal advancement in molecular medicine, offering a powerful tool for modulating gene expression with precision and specificity. Through targeted intervention in genetic pathways, ASOs provide a therapeutic strategy for a myriad of diseases that were previously considered intractable at the genetic level. The design of ASOs involves a sophisticated array of strategies, each tailored to maximize efficacy, specificity, stability, and safety, and minimize off-target effects and toxicity.
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