Michael Schneider - Springfield IL, US Andrew Wilber - Springfield IL, US
International Classification:
A61K048/00 A61K009/127 C12N015/88 A61K038/21
US Classification:
514/044000, 435/458000, 424/450000, 424/085600
Abstract:
Deoxyribonuclase 1-like 3 (D3) hydrolyzes lipid-complexed DNA and decreases transfection efficiency in liposomal transfection (lipofection) Systems. Accordingly, D1L3 provides a more accurate test of the efficiency of lipid/liposomal based gene therapy than current standards using deoxyribonuclease 1 (D1). Moreover, it has been found that mice with systemic lupus erythematosus (lupus) have lowered D1L3 activity. Therefore, differing therapeutic benefits may result from either the upward or downward therapeutic regulation of D1L3 activity. For example, blocking D1L3 activity enhances liposomal transfection for gene therapy, while increasing D1L3 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.
Andrew WILBER - Rochester IL, US - Springfield IL, US
International Classification:
C12N 15/85 C12N 15/63
Abstract:
An inducible expression cassette for controlled expression of multiple genes includes the DNA sequences of a bidirectional promoter inserted between two DNA sequences. Transfection into a host cell and the two DNA sequences encode genes of interest, provides one DNA sequence expressed constitutively and manipulation of the expression of the other DNA sequence. A regulatory DNA cassette containing another bidirectional promoter and two DNA sequences that encode a marker and a regulatory expression product is also disclosed. Both cassettes can be incorporated in a non-viral vector, like the Sleeping Beauty transposon, or a viral vector to induce controlled expression of multiple genes into host cells. A kit containing a package of each above vector type is also disclosed, as is a method of transforming a host cell.