ABSTRACT
Antisense oligonucleotides (ASOs) are designed to modulate the information transfer from gene to protein-in essence to alter mRNA intermediary metabolism. mRNA intermediary metabolism is extremely complex beginning with transcription and concluding with degradation usually after translation. Each step is complex and in dynamic equilibrium with competing pathways. Although great progress has been made in understanding these processes, much remains unknown, and we
have only begun to understand the potential impacts of ASOs on these processes and the factors that influence the outcomes.
