Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics.

ABSTRACT: Oligonucleotide therapeutics are a novel promising class of drugs designed to specifically target either coding or non-coding RNA molecules to revolutionize treatment of various diseases. During preclinical development, investigations of the pharmacokinetic characteristics of these oligonucleotide-based drug candidates are essential. Oligonucleotides possess a long history of chemical modifications to enhance their stability and binding affinity, as well as reducing toxicity. Phosphorothioate backbone modifications of oligonucleotides were a hallmark of this development process that greatly enhanced plasma stability and protein binding of these agents. Modifications such as 2'-O-methylation further improved stability, while other modifications of the ribose, such as locked nucleic acid (LN

SUBMITTER: Herkt M 

PROVIDER: S-EPMC7854291 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature