Characterization of Cationic Bolaamphiphile Vesicles for siRNA Delivery into Tumors and Brain.

ABSTRACT: Small interfering RNAs (siRNAs) are potential therapeutic substances due to their gene silencing capability as exemplified by the recent approval by the US Food and Drug Administration (FDA) of the first siRNA therapeutic agent (patisiran). However, the delivery of naked siRNAs is challenging because of their short plasma half-lives and poor cell penetrability. In this study, we used vesicles made from bolaamphiphiles (bolas), GLH-19 and GLH-20, to investigate their ability to protect siRNA from degradation by nucleases while delivering it to target cells, including cells in the brain. Based on computational and experimental studies, we found that GLH-19 vesicles have better delivery characteristics than do GLH-20 vesicles in terms of stability, binding affinity, protection against nucleas

SUBMITTER: Kim T 

PROVIDER: S-EPMC7090283 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature