siRNA Delivery to Inhibit Tumor Growth in the Central Nervous System
siRNA Delivery to Inhibit Tumor Growth in the Central Nervous System
This invention provides compositions and methods that utilize a cell-permeable complex for facilitating the delivery of a double-stranded RNA into the central nervous system (CNS) to reduce the expression of a target protein, and in turn inhibit the growth of a tumor. Specifically, by convection-enhanced delivery to the CNS, a cell-permeable complex comprises a double stranded RNA that is effective in inhibiting the expression of the ta...
New York, NY, United States
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Background
Brain Tumor Drug Delivery Inhibited by Slow Diffusion Drug Distribution The blood-brain barrier prevents the delivery of many systemically administered molecules to the brain. Current methods to improve drug delivery to the brain have had some success but ultimately have been clinically inadequate, mainly because that those delivery methods rely on diffusion to distribute the compound throughout the tissue, which is slow and inefficient in brain tissue. A convection-enhanced regional drug delivery method was developed which uses a positive-pressure, microinfusion mechanism to produce convective forces to distribute a therapeutic agent throughout the brain. 

RNA interference (RNAi) is an endogenous cellular mechanism that down-regulates genes post-transcriptionally in a very specific and efficient way. siRNA alone does not cross the blood brain barrier and has poor uptake by cells. The successful delivery of siRNA to the neurons of the brain tissue is the first challenge for developing its potential as a therapeutic tool. 

Cell-Permeable Complex Facilitates Drug Delivery and Inhibits Tumor Growth This invention provides compositions and methods that utilize a cell-permeable complex for facilitating the delivery of a double-stranded RNA into the central nervous system (CNS) to reduce the expression of a target protein, and in turn inhibit the growth of a tumor. Specifically, by convection-enhanced delivery to the CNS, a cell-permeable complex comprises a double stranded RNA that is effective in inhibiting the expression of the target protein, operably linked to a cell penetrating peptide. Consequently, inhibition of the target protein will induce apoptosis in tumor cells. 

Applications: 
• Inhibiting the growth of a tumor in the CNS 
• Preventing cell death associated with ischemia in the CNS 
• Useful for inhibiting inflammation in the CNS 

Advantages: 
• The penetratin 1-siRNA provides is less toxic while delivering siRNA to neurons 
• The cell-penetrating peptide in the complex typically function with high efficiency, even at low concentrations 
 
Lead Inventors: 
Carol M. Troy, M.D., Ph.D.; Giselle F. Prunell; E. Sander Connolly; Andrew F. Ducruet 
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