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1. (WO2003072741) THERAPEUTIC REGULATION OF DEOXYRIBONUCLEASE-1-LIKE-3 ACTIVITY

Pub. No.:    WO/2003/072741    International Application No.:    PCT/US2003/005654
Publication Date: Sep 4, 2003 International Filing Date: Feb 26, 2003
IPC: A61K 48/00
C12N 9/22
Applicants: SOUTHERN ILLINOIS UNIVERSITY
SCHNEIDER, Michael, C.
WILBUR, Andrew
Inventors: SCHNEIDER, Michael, C.
WILBUR, Andrew
Title: THERAPEUTIC REGULATION OF DEOXYRIBONUCLEASE-1-LIKE-3 ACTIVITY
Abstract:
Deoxyribonuclease 1-like 3 (D3) hydrolyzes lipid-complexed DNA and decreases transfection efficiency in liposomal transfection (lipofection) systems. Accordingly, D1 L3 provides a more accurate test of the efficiency of lipid/liposomal based gene therapy than current standards using deoxyribonuclease 1 (Dl). Moreover, it has been found that mice with systemic lupus erythematosus (lupus) have lowered D1 L3 activity. Therefore, differing therapeutic benefits may result from either the upward or downward therapeutic regulation of D1 L3 activity. For example, blocking D1 L3 activity enhances liposomal transfection for gene therapy, while increasing D 1 L3 activity may enhance destruction of pathogenic DNA, whether viral, bacterial or endogenous. Destruction of pathogenic DNA may provide treatment for lupus, or viral and oncogenic diseases.