Liver-specific gene expression cassettes, and methods of use
AssigneesThe Board of Trustees of the Leland Stanford Junior University, University of Washington
InventorsCarol H. Miao, Mark A. Kay
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Abstract
In one aspect, the present invention provides nucleic acid expression cassettes that are predominantly expressed in the mammalian liver. The present invention also provides vectors comprising a nucleic acid expression cassette that is predominantly expressed in the mammalian liver. The present invention also provides methods of ameliorating the symptoms of a disease, the methods including the steps of introducing into the liver of a mammalian subject a vector comprising a nucleic acid expression cassette that encodes a polypeptide, and expressing a therapeutic amount of the polypeptide in the liver.
Claims8
A nucleic acid expression cassette capable of expressing human Factor IX, wherein the cassette is predominantly expressed in the mammalian liver, said cassette comprising:
- (a) a hepatic locus control element consisting of SEQ ID NO:4 or SEQ ID NO:9;
- (b) a heterologous hepatic promoter located 3′ to the hepatic locus control element, said promoter consisting of SEQ ID NO:
- 5;
- (c) a Factor IX coding sequence located 3′ to the hepatic promoter, said coding sequence comprising SEQ ID NO:2;
- (d) a polyadenylation signal located 3′ to the intron sequence, said polyadenylation signal consisting of SEQ ID NO:6;
- and
- (e) an intron located 3′ to the hepatic promoter and 5′ to the polyadenylation signal, wherein said intron consists of SEQ ID NO:1,
- wherein elements (a), (b), (c), (d) and (e) are operably linked to express the polypeptide encoded by the coding sequence.
A nucleic acid expression cassette capable of expressing human Factor IX, wherein the cassette is predominantly expressed in the mammalian liver, said cassette comprising:
- (a) a hepatic locus control element consisting of SEQ ID NO:4 or SEQ ID NO:9;
- (b) a hepatic promoter located 3′ to the hepatic locus control element, said promoter consisting of SEQ ID NO:5;
- (c) a Factor IX coding sequence located 3′ to the hepatic promoter, said coding sequence comprising SEQ ID NO:2;
- (d) a polyadenylation signal located 3′ to the intron sequence, said polyadenylation signal consisting of SEQ ID NO:6;
- and
- (e) an intron located 3′ to the hepatic promoter and 5′ to the polyadenylation signal, wherein said intron consists of SEQ ID NO:1, and
- (f) an untranslated region located 3′ to the coding region and the intron, said untranslated region consisting of SEQ ID NO:7;
- wherein elements (a), (b), (c), (d) and (e) are operably linked to express the polypeptide encoded by the coding sequence.
Dependencies are inferred from the claim text.