Modulation of the blood-brain barrier for therapeutic benefit

Abstract

To date this lab has carried out novel work in the area of low-molecular weight (<1 kDa) drug delivery to the retina (Campbell, Nguyen et al. 2009; Tam, Kiang et al. 2010). This work was carried by suppression of claudin-5 (CL5), a tight junction protein expressed on endothelial cells of the inner blood-retina barrier, by delivery of small interfering RNA molecules. Recently this work has been extended to include a limited study on suppression of claudin-5 at the blood-brain barrier (BBB), and this resulted in successful delivery of a low-molecular weight compound to the brain (Campbell, Humphries et al. 2011). The primary goal of the study presented here is to extend this BBB modulation to a comprehensive exploration of the benefits of barrier modulation to conditions affecting the brain.