Template-independent enzymatic synthesis of RNA oligonucleotides
Technology tamfitronics MainSynthesis of RNA oligonucleotides by the phosphoramidite chemical method has enabled many valuable discoveries and new ways to treat disease throughout the past 50 years1,2,3,4. This has culminated in the development of an array of therapeutic modalities that include antisense oligonucleotides (ASOs) and short interfering RNA (siRNA)5,6,7. ASOs and siRNA have traditionally been used to treat rare diseases such as spinal muscular atrophy and hereditary transthyretin-mediated amyloidosis8,9,10. They are often chemically modified, which offers therapeutic advantages such as increased binding affinity, stability, and protection from nuclease degradation4,11. More recently, the N-acetylgalactosamine (GalNAc) ligand conjugated to siRNA enabled tissue-specific delivery of the active RNA drug12,13. These advances have resulted in unparalleled growth in the...
