Expression of factor IX in gene therapy vectors

Chemistry: molecular biology and microbiology – Vector – per se

Reexamination Certificate

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C435S069600, C536S023500

Reexamination Certificate

active

08030065

ABSTRACT:
Two mechanisms are provided for improving the expression of Factor IX in gene therapy vectors. The first is the use of a specific Factor IX polynucleotide coding sequence designed for optimal expression. The second is the use of transcriptional regulatory regions minimized in size so that they can be used to express Factor IX, as well as any other gene of interest, in a size-constrained environment such as in a self complementary gene therapy vector system.

REFERENCES:
patent: 6093392 (2000-07-01), High et al.
patent: 6670176 (2003-12-01), Samulski et al.
patent: 2002/0064812 (2002-05-01), Connelly et al.
patent: 2003/0022378 (2003-01-01), Ehrhardt et al.
patent: 2003/0077812 (2003-04-01), McArthur et al.
patent: 2004/0009151 (2004-01-01), Kay et al.
patent: 2004/0023333 (2004-02-01), Hauser et al.
patent: 2004/0029106 (2004-02-01), Samulski et al.
patent: WO 01/66149 (2001-09-01), None
patent: WO 01/92551 (2001-12-01), None
patent: WO 01/98482 (2001-12-01), None
patent: WO 02/064799 (2002-09-01), None
Haas et al., Current Biology (1996), vol. 6, No. 3, pp. 315-324.
Wang,Z. et al.; “Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo”, Gene Therapy 10:2105-2111 (2003).
McCarty, D.M. et al.; “Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo”, Gene Therapy 10:2112-2118 (2003).
McCarty, D.M. et al.; “Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction indepedently of DNA synthesis”, Gene Therapy 8:1248-1254 (2001).
Ziegler, R.J., et al.; “AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice”, Mol. Therap. 9(2):231-240 (2004).
Miao, C.H., at al., “Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stablizes hepatic factor IX gene expression in vivo but not in vitro”, Mol Ther 1(6):522-532 (2000).
Nakai, H., et al, “Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes are Primarily Responsible for Stable Liver Transduction In Vivo”;Journal of Virology, vol. 75, No. 15, Aug. 1, 2001, pp. 6969-6976.
Database Accession No. AX249946, “Sequence 4 from Patent WO0166149”, Sep. 13, 2001; E.B.I. Hinxton U.K., XP002550494.
Database Accession No. ABS68099, Nov. 19, 2002, “Clotting Factor IX Construct pFIXABCD”, Seldon, R.F., et al., XP002550495.
Database Accession No. AAI71010, Dec. 27, 2001, “Human Apolipoprotein E Gene Hepatic Locus Control Element”, E.B.I. Hinxton U.K., XP002550496.
Database Accession No. AAI71009, Mar. 18, 2002, “Human Apolipoprotein E Gene Enhancer”, C.H. Miao et al., XP002550497.
Database Accession No. AAI71006, Mar. 18, 2002, “Human Alpha1 Antitrypsin Promoter”, E.B.I. Hinxton U.K., XP002550498.

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