Oligonucleotide therapeutics aim to treat disease by altering gene expression. They work by modulating gene expression through antisense or antigene technology to complementarily bind to RNA or DNA, respectively. Successful oligonucleotide therapy requires overcoming challenges such as nuclease degradation and poor cellular uptake. Various delivery strategies including liposomes, dendrimers, polymers, and conjugation to carrier peptides are being explored to protect oligonucleotides and improve cellular delivery, though none have achieved full success yet. Overall, oligonucleotide therapeutics show promise if these delivery challenges can be addressed.
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