Method for opening tight junctions

摘要

The present invention is directed to a method and use of RNA interference (RNAi) for the transient, reversible and controlled opening of the tight junctions of the blood brain barrier and/or the blood retinal barrier. This method may be used in the treatment of many diseases and disorders which require the opening of the blood brain barrier and/or blood retinal barrier. Such methods generally involve the use of an RNAi-inducing agent, such as siRNA, miRNA, shRNA or an RNAi-inducing vector whose presence within a cell results in production of an siRNA or shRNA, targeting tight junction proteins to open the blood brain barrier and/or blood retinal barrier.

主权项

1. A method for the treatment of a traumatic brain injury or stroke wherein the treatment comprises the systemic or viral mediated delivery of a RNAi inducing agent targeting claudin-5 to allow the permeation and free diffusion of water across the blood brain barrier, and results in the reversible and transient RNAi-mediated suppression of claudin-5 in brain capillary endothelial cells and wherein RNAi-mediated suppression commences from approximately 24 hours post delivery of the RNAi inducing agent and lasts up to approximately 72 hours post delivery of the RNAi inducing agent.