A novel peptide ligand-coated nano-siRNA-lipoplex technology for kidney targeted gene therapy.

ABSTRACT:

Objectives

Small interfering RNA (siRNA) that silences specific disease-related genes holds the promise for the treatment of renal disease. However, delivery to the intended site of action remains a major obstacle. The goal of this study was to develop glomerulus-specific siRNA particles for targeted gene therapy of kidney diseases.

Methods

We used a novel nanoparticle-based system comprised of siRNA in cationic liposomes (Lip) coated with non-inhibitory plasminogen activator inhibitor 1R (PAI-1R) that selectively targets glomerular cells and tested it with transforming growth factor-beta 1 (TGF-β1)-siRNA in nephritic rat model.

Results

At the optimized ratio of components, three of PAI-1R, Lip and siRNA formed the compact nanostructured particles with close to neutra

SUBMITTER: Liu X 

PROVIDER: S-EPMC9641478 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature