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Abstract
Grant Number: 2R44GM074292-02 Project Title: Amphiphilic Polycations for Nucleic Acid Delivery
PI Information: Name Title ROZEMA, DAVID B. dave.rozema@mirusbio.com Abstract: DESCRIPTION (provided by applicant): Given its central role in metabolism, the secretion of circulating proteins, and infectious disorders, a large number of genetic or acquired disorders could be corrected by liver-directed delivery of nucleic acids to instill beneficial functions or inhibit deleterious ones. However, a safe and effective delivery method for transferring nucleic acids molecules to hepatocytes remains to be perfected. To date, most in vivo nucleic acid delivery vehicles have been colloid particles with size grater than 50 nm. We believe that we have found an innovative strategy for the delivery of molecular conjugates of siRNA. This formulation relies upon several new innovations: the development-as a result of Phase I research- of membrane lytic polymers, the ability to reversibly mask the positive charges of these polymers until they reach an acidic environment such as the endosome/lysosome, the ability to target these masked membrane lytic polymers to hepatocytes in vivo, and the formation of siRNA-polymer conjugates that like polymers can be targeted to hepatocytes in vivo.
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Institution: MIRUS BIO CORPORATION 505 SOUTH ROSA ROAD, STE 104 MADISON, WI 537191264 Fiscal Year: 2007 Department: Project Start: 01-SEP-2005 Project End: 31-MAR-2009 ICD: NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES IRG: ZRG1