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Chemistry, structure and function of approved oligonucleotide therapeutics
1. Introduction: In the last 25 years, 18 oligonucleotide therapeutics have been approved for treating diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and hereditary transthyretin-mediated amyloidosis. These therapies operate through different mechanisms, including antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi), and RNA aptamers. 2. Historical Background: Oligonucleotide therapeutics trace back to the 1950s and 1960s with early discoveries in DNA and RNA synthesis. Pioneers like Todd, Khorana, …
