Patent ID: 7316925
Filing Date: 2008-01-08
Classification: A61K,A61P,C07K,C12N

Abstract:
1. A synthetic mammalian expression plasmid for plasmid mediated gene supplementation in a species comprising: (a) a synthetic or eukaryotic promoter comprising SEQ ID NO: 15; (b) a codon-optimized eukaryotic therapeutic gene sequence comprising SEQ ID NO: 7; (c) a polyadenylation signal; (d) a selectable marker gene sequence; (e) a ribosomal binding site; (f) a promoter for the selectable marker gene sequence; and (g) an origin of replication; wherein the synthetic or eukaryotic promoter, the codon-optimized-eukaryotic therapeutic gene sequence, and the polyadenylation signal comprise therapeutic elements of the synthetic mammalian expression plasmid; the therapeutic elements are operatively linked and located in a first operatively-linked arrangement; the selectable marker gene promoter, the ribosomal binding site, the selectable marker gene sequence, and the origin of replication comprise replication elements of the synthetic mammalian expression plasmid; the replication elements are operatively linked and located in a second-operatively-linked arrangement; and the first-operatively linked arrangement and the second-operatively-linked arrangement comprise a circular structure of the synthetic mammalian expression plasmid.