Peptide- and Amine-Modified Glucan Particles for the Delivery of Therapeutic siRNA.

ABSTRACT: Translation of siRNA technology into the clinic is limited by the need for improved delivery systems that target specific cell types. Macrophages are particularly attractive targets for RNAi therapy because they promote pathogenic inflammatory responses in a number of important human diseases. We previously demonstrated that a multicomponent formulation of β-1,3-d-glucan-encapsulated siRNA particles (GeRPs) can specifically and potently silence genes in mouse macrophages. A major advance would be to simplify the GeRP system by reducing the number of delivery components, thus enabling more facile manufacturing and future commercialization. Here we report the synthesis and evaluation of a simplified glucan-based particle (GP) capable of delivering siRNA in vivo to selectively silence macroph

SUBMITTER: Cohen JL 

PROVIDER: S-EPMC5153885 | biostudies-literature | 2016 Mar

REPOSITORIES: biostudies-literature