02172oam 2200325z- 450 991055519480332120210108010103.01-119-07030-91-119-07029-51-119-07015-5(CKB)4330000000008301(MiAaPQ)EBC5452560(EXLCZ)99433000000000830120180812cuuuuuuuu -u- -engOligonucleotide-based drugs and therapeutics preclinical and clinical considerations for development /edited by Nicolay Ferrari, Rosanne SeguinJohn Wiley & Sons, Inc1-118-53733-5 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.OligonucleotidesTherapeutic useAntisense nucleic acidsTherapeutic useOligonucleotidesTherapeutic use.Antisense nucleic acidsTherapeutic use.572.8/5Ferrari Nicolay1969-Seguin RosanneBOOK9910555194803321Oligonucleotide-Based Drugs and Therapeutics2815752UNINA