Lipid nanoparticle delivery of a microRNA-145 inhibitor improves experimental pulmonary hypertension.

ABSTRACT: Therapies that exploit RNA interference (RNAi) hold great potential for improving disease outcomes. However, there are several challenges that limit the application of RNAi therapeutics. One of the most important challenges is effective delivery of oligonucleotides to target cells and reduced delivery to non-target cells. We have previously developed a functionalized cationic lipopolyamine (Star:Star-mPEG-550) for in vivo delivery of siRNA to pulmonary vascular cells. This optimized lipid formulation enhances the retention of siRNA in mouse lungs and achieves significant knockdown of target gene expression for at least 10days following a single intravenous injection. Although this suggests great potential for developing lung-directed RNAi-based therapies, the application of Star:Star-mPEG

SUBMITTER: McLendon JM 

PROVIDER: S-EPMC4477514 | biostudies-literature | 2015 Jul

REPOSITORIES: biostudies-literature