1Becker J, Schwab R, Moller-Taube A, et al. Characterization of the facter Ⅷ defect in 147 patients with sporadic hemophilia A: family studies indicate a mutation type-dependent sex ratio of mutation frequencies[J]. Am J Hum Genet, 1996,58(4):657.
2Ragni MV, et al. 1986 update of HIV seroprevalence, serroconversion,AIDS incidence,and immundogic correlates of HIV infection in patients with the mophilia A and B[J]. Blood, 1987, 70: 786-790.
3Yohshitakes S, Schach BG, Foster DG, et al. Nucleotide sequence of the gene for human factor Ⅸ (anti-hemophiliac factor B) [J]. Biochemistry, 1982,24(14) : 3736-3750.
4Dwarki VJ, Belloni P, Nijjar T, et al. Gene Therapy for Hemophilia A: production of therapeutic levels of human factor Ⅷ in vivo in mice[J]. Proc Natl Acad Sci USA , 1995,92(4):1023.
5Dela SH, et al. Active carboxylated human fact Ⅸ expressed using recombinant DNA techniques[J]. Nature, 1985,316: 268.
6Vanden DT, et al. Oncoretroviral and lentiviral vecter-medimted gene therapy[J]. Methods Enzymol, 2002,346: 572-589.
7Vanden DT,Vanslembrouek V,Goovaerts I,et al. Long-term expression of human coagulation faeter Ⅷ and correetion of hemophilia A after in vivo retroviral gene transfer in faeter Ⅷ-defieient miee[J]. Proe Natl Aead Sei USA, 1999,96(18) : 10379-10384.
8Balangue C, Zhou J, Dai Y, et al. Sustained high-level expression of full-length human facter Ⅷ and restoration of.clotting activity in hemophilic mice using a minimal adenovirus vector[J]. Blood, 2000,95 (3): 820-828.
9Chuah MK, Schiedner G, Thorrez L, et al. Therapeutic. facter Ⅷ levels and negligible toxicity in mouse and dog model of hemophilia A following gene therapy with high-capacity advenovial vectors[J]. Blood,2003,101(5) :1734-1742.
10Chao H, Mao L,Bruce AT,et al. Sustained expression of human facter Ⅷ in mice using a parvovirus-based vector [J]. Blood, 2000,95(5) : 1594-1599.
2Jallat S.Perraud F,Dalemans W,et al.Characterization of recombinant human factor IX expressed in transgenic mice and in derived trans-immortalized hepatic cell lines.EMBO J,1990,9 (10):3295-3301.
3Kaleko M,Kayda D,Sakhuja K,et al.Genomic sequences increase adenoviral vector-mediated factor IX expression 1,900 fold and enable sustained expression in vivo.J.Cell.Biochem,1995,21,A366.
4Chen SH,Schoof JM,Weinmann AF,et al.Heteroduplex screening for molecular defects in factor IX genes from haemophilia B families.Br J Haematol,1995,89(2):409-412.
5Miao CH,Ohashi K,Patijn GA,et al.Inclusion of the hepatic locus control region,an intron,and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro.Mol ther,2000,1(6):522-532.
6Miao CH,Thompson AR,Loeb K,et al.Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo.Mol ther,2001,3 (6):947-957.
7Viiala NO,Larsen SR,Rasko JE.Gene therapy for hemophilia:clinical trials and technical tribulations.Semin Thromb Hemost,2009,35(1):81-92.
