By contrast, conventional small-molecule pharmaceuticals require much larger, and often iterative, screening efforts followed by extensive medicinal chemistry optimization. In addition, the use of oligonucleotides allows for precision and/or personalized medicine approaches as they can theoretically be designed to selectively target any gene with minimal, or at least predictable, off-target effects.
Highly specific lead compounds can often be rationally designed based on knowledge of the primary sequence of a target gene alone and lead candidates identified by rapid screening. The majority of oligonucleotide modalities interact with their cognate target molecules via complementary Watson–Crick base pairing, and so interrogation of the putative target sequence is relatively straightforward. Although the majority of oligonucleotide therapeutics have focused on gene silencing, other strategies are being pursued, including splice modulation and gene activation, expanding the range of possible targets beyond what is generally accessible to conventional pharmaceutical modalities. Oligonucleotides are nucleic acid polymers with the potential to treat or manage a wide range of diseases.
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