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Last Updated: September 23, 2020

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Claims for Patent: 8,628,956

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Summary for Patent: 8,628,956
Title:Expression vector suitable for expression of a coding sequence for gene therapy
Abstract: Provided is an expression vector for gene therapy having a novel combination of transcriptional regulatory elements, including a promoter, an enhancer, an intron, an untranslated region (UTR) and a locus control region (LCR). The expression vector enables sustained expression of a liver tissue-specific gene, and thus, can be effectively used for treating thrombosis, hemophilia, liver cancer, etc.
Inventor(s): Koh; Daekyung (Yongin-si, KR), Lee; Kyuhyun (Yongin-si, KR), Lee; Hyeon (Yongin-si, KR), Yun; Seongtae (Yongin-si, KR), Jo; Eui-Cheol (Yongin-si, KR)
Assignee: Mogam Biotechnology Research Institute (Yongin-Si, KR)
Application Number:13/423,474
Patent Claims:1. An isolated polynucleotide consisting of the nucleotide sequence as set forth in SEQ ID NO: 49.

2. An expression cassette comprising: a promoter; a coding sequence operably linked to and under control of the promoter; and an intron consisting of the nucleotide sequence as set forth in SEQ ID NO: 49, said intron being operably linked to the coding sequence.

3. The expression cassette of claim 2, wherein the promoter is selected from the group consisting of polynucleotides having the nucleotide sequences as set forth in SEQ ID NOS: 41, 42 and 43.

4. The expression cassette of claim 2, further comprising at least one of enhancers having the nucleotide sequences as set forth in SEQ ID NOS: 44 or 45, wherein the at least one enhancers are operably linked to the coding sequence.

5. The expression cassette of claim 2, further comprising at least one of locus control regions having the nucleotide sequences selected from the group consisting of the nucleotide sequences as set forth in SEQ ID NOS: 58-60 and 61, wherein the at least one locus control regions are operably linked to the coding sequence.

6. The expression cassette of claim 2, further comprising untranslated regions having nucleotide the sequences of SEQ ID NOS: 62 and 63 at 5' and 3'-ends of the coding sequence, respectively, wherein the untranslated regions are operably linked to the coding sequence.

7. The expression cassette of claim 2, wherein the coding sequence is a nucleotide sequence encoding a liver-specific protein selected from the group consisting of albumin, .alpha.-fetoprotein, .alpha.-glucosidase, .alpha.1-antitrypsin, antithrombin, lipoproteins, ceruloplasmin, coagulation factor VII, coagulation factor VIII, coagulation factor IX, erythropoietin, fibrinogen, glucocerebrosidase, haptoglobin, IGF-1, insulin, plasminogen, prothrombin and transferrin.

8. An expression vector comprising the expression cassette of claim 2.

9. The expression cassette of claim 4, further comprising at least one of locus control regions having the nucleotide sequences selected from the group consisting of the nucleotide sequences as set forth in SEQ ID NOS: 58-60 and 61, wherein the at least one locus control regions are operably linked to the coding sequence.

10. The expression cassette of claim 9, further comprising untranslated regions, having the nucleotide sequences of SEQ ID NOS: 62 and 63 at 5' and 3'-ends of the coding sequence, respectively, wherein the untranslated regions are operably linked to the coding sequence.

11. The expression cassette of claim 4, further comprising untranslated regions having the nucleotide sequences of SEQ ID NOS: 62 and 63 at 5' and 3'-ends of the coding sequence, respectively, wherein the untranslated regions are operably linked to the coding sequence.

12. The expression cassette of claim 5, further comprising untranslated regions having the nucleotide sequences of SEQ ID NOS: 62 and 63 at 5' and 3'-ends of the coding sequence, respectively, wherein the untranslated regions are operably linked to the coding sequence.

13. An expression vector comprising the expression cassette of claim 4.

14. An expression vector comprising the expression cassette of claim 5.

15. An expression vector comprising the expression cassette of claim 6.

16. An expression vector comprising the expression cassette of claim 9.

17. An expression vector comprising the expression cassette of claim 10.

18. An expression vector comprising the expression cassette of claim 11.

19. An expression vector comprising the expression cassette of claim 12.

Details for Patent 8,628,956

Applicant Tradename Biologic Ingredient Dosage Form BLA Number Approval Date Patent No. Assignee Estimated Patent Expiration Status Orphan Source
Schering INTRON A interferon alfa-2b VIAL 103132 001 1986-06-04   Start Trial Mogam Biotechnology Research Institute (Yongin-Si, KR) 2028-02-14 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 002 1986-06-04   Start Trial Mogam Biotechnology Research Institute (Yongin-Si, KR) 2028-02-14 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 003 1986-06-04   Start Trial Mogam Biotechnology Research Institute (Yongin-Si, KR) 2028-02-14 RX search
>Applicant >Tradename >Biologic Ingredient >Dosage Form >BLA >Number >Approval Date >Patent No. >Assignee >Estimated Patent Expiration >Status >Orphan >Source

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