METHOD FOR OPENING TIGHT JUNCTIONS
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Abstract
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.
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Citations
53 Claims
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1-36. -36. (canceled)
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37. A method for the treatment of a disease or disorder selected from a neurodegenerative disorder, a neuropsychiatric disorder, brain tumor, and retinal disorder, the method comprising the reversible, transient and controlled RNAi-mediated size selective opening of the paracellular pathway of the blood brain barrier wherein the method comprises:
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identifying a subject at risk for developing the disease or disorder; administering an effective amount of an RNAi inducing agent targeting tight junction proteins selected from occludin, claudin 1-19 or 21 by delivery of the RNAi inducing agent to result in the transient and reversible RNAi-mediated suppression of blood brain barrier tight junction protein transcripts in brain capillary endothelial or retinal endothelial cells and allow the permeation of active agents used in the treatment of the disease or disorder to the brain capillary endothelial and/or retinal cells; and administering an active agent suitable for the treatment of the disease or disorder. - View Dependent Claims (38, 39, 40, 41, 42, 43, 44, 45, 46, 47, 48, 49, 50, 51, 52, 53)
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Specification