Mechanisms of oligonucleotide actions -- The medicinal chemistry of antisense oligonucleotides -- Cellular pharmacology of antisense oligonucleotides -- Pharmacokinetics and pharmacodynamics of antisense oligonucleotides -- Tissue distribution, metabolism and clearance -- Hybridization-independent effects : principles and specific considerations for oligonucleotide drugs -- Hybridization-dependent effects : the prediction, evaluation and consequences of unintended target hybridization -- Class-related proinflammatory effects -- Exaggerated pharmacology -- Genotoxicity tests for novel oligonucleotide-based therapeutics -- Reproductive and developmental toxicity testing strategies for oligonucleotide-based therapeutics -- Specific considerations for preclinical development of inhaled oligonucleotides -- Lessons learned in oncology programs -- Inhaled antisense for treatment of respiratory disease -- Antisense oligonucleotides for treatment of neurology diseases -- Nucleic acids as adjuvants -- Splice-switching oligonucleotides -- CMC aspects for the clinical development of spiegelmers. |