Kinetic Modeling to Accelerate the Development of Nucleic Acid Formulations.

ABSTRACT: A critical hurdle in the clinical translation of nucleic acid drugs is the inefficiency in testing formulations for therapeutic potential. Specifically, the ability to quantitatively predict gene expression is lacking when transitioning between cell culture and animal studies. We address this challenge by developing a mathematical framework that can reliably predict short-interfering RNA (siRNA)-mediated gene silencing with as few as one experimental data point as an input, evaluate the efficacies of existing formulations in an expeditious manner, and ultimately guide the design of nanocarriers with optimized performances. The model herein consisted of only essential rate-limiting steps and parameters with easily characterizable values of the RNA interference process, enabling the easy ide

SUBMITTER: Roh EH 

PROVIDER: S-EPMC8860063 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature