Gene Therapy of Hemophilia B

• References

• 1 Anderson WF. Human gene therapy. Science 1992; 256: 808-13
• 2 Miller AD. Human gene therapy comes of age. Nature 1992; 357: 455-66
• 3 McKee PA. Hemostasis and disorders of blood coagulation. In: The Metabolic Basis of Inherited Diseases, 5th Ed. Stanbury JB, Wyn-gaarden JB, Frederickson DS, Goldstein JL, Brown MS. (eds). McGraw-Hill; New York: 1983. pp 1531-60
• 4 Kurachi K, Kurachi S, Furukawa M, Yao SN. Biology of factor IX. Semin Thromb Hemostas, in press.
• 5 Evans JP, Brinkhous KM, Brayer GD, Reisner HM, High KA. Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Natl Acad Sci USA 1989; 86: 10095-9
• 6 White 2d GC, Blatt PM, McMillan CW, Webster WP, Lesesne HR, Roberts HR. Medical complications of hemophilia. South Med J 1980; 73: 155-60
• 7 Yoshitake S, Schach BG, Foster DC, Davie EW, Kurachi K. Nucleotide sequence of the gene for human factor IX (antihemophilic factor B). Biochemistry 1985; 24: 3736-50
• 8 Eglitis MA, Anderson WF. Retroviral vectors for introduction of genes into mammalian cells. BioTechniques 1988; 6: 608-14
• 9 Ferry N, Duplessis O, Houssin D, Danos O, Heard JM. Retroviral-mediated gene transfer into hepatocytes in vivo . Proc Natl Acad Sci USA 1991; 88: 8377-81
• 10 Chowdhury JR, Grossman M, Gupta S, Chowdhury NR, Baker Jr JR, Wilson JM. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science 1991; 254: 1802-5
• 11 Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T, Finegold M, Darlington G, Pokorny W, et al. Expression of human αl-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 1992; 89: 89-93
• 12 Bovsun M. Gene therapy: New protocols, risks, and possible advances. ASM News 1993; 59: 54-8
• 13 Rosenfeld MA, Siegfried W, Yoshimura K, Yoneyama K, Fukayama M, Stier LE, Paakko PK, Gilardi P, Stratford-Perricaudet LD, Perricaudet M, Jallat S, Pavirani A, Lecocq JP, Crystal RG. Adenovirus-mediated transfer of a recombinant al-antitrypsin gene to the lung epithelium in vivo . Science 1991; 252: 431-4
• 14 Quantin B, Perricaudet LD, Tajbakhsh S, Mandel JL. Adenovirus as an expression vector in muscle cells in vivo . Proc Natl Acad Sci USA 1992; 89: 2581-4
• 15 Wagner E, Zatloukal K, Cotten M, Kirlappos H, Mechtler K, Curiel DT, Birnstiel ML. Coupling of adenovirus to transferrin-poly-lysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc Natl Acad Sci USA 1992; 89: 6099-103
• 16 Nabel GJ. Immunotherapy of malignancy by in vivo gene transfer into tumors (protocol). Human Gene Therapy 1992; 3: 399-410
• 17 Hermonat PL, Muzyczka N. Use of adeno-associated virus as a mammalian DNA cloning vector: Transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci USA 1984; 81: 6466-70
• 18 Kotin RM, Siniscalco M, Samulski RJ, Zhu X, Hunter L, Laughlin CA, McLaughlin S, Muzyczka N, Rocchi M, Berns KI. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 1990; 87: 2211-5
• 19 Louis St. D, Verma IM. An alternative approach to somatic cell gene therapy. Proc Natl Acad Sci USA 1988; 85: 3150-4
• 20 Palmer TD, Thompson AR, Miller AD. Production of human factor IX in animals by genetically modified skin fibroblasts: Potential therapy for hemophilia B. Blood 1989; 73: 438-45
• 21 Scharfmann R, Axelrod JH, Verma IM. Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc Natl Acad Sci USA 1991; 88: 4626-30
• 22 Yao SN, Wilson JM, Nabel EG, Kurachi S, Hachiya H, Kurachi K. Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B. Proc Natl Acad Sci USA 1991; 88: 8101-5
• 23 Armentano D, Thompson AR, Darlington G, Woo SL. Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: Potential for gene therapy of hemophilia B. Proc Natl Acad Sci USA 1990; 87: 6141-5
• 24 Ponder KP, Gupta S, Leland F, Darlington G, Finegold M, DeMayo J, Ledley FD, Chowdhury JR, Woo SLC. Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation. Proc Natl Acad Sci USA 1991; 88: 1217-21
• 25 Gussoni E, Pavlath GK, Lanctot AM, Sharma KR, Miller RG, Steinman L, Blau HM. Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 1992; 356: 435-8
• 26 Huard J, Bouchard JP, Roy R, Malouin F, Dansereau G, Labrecque C, Albert N, Richards CL, Lemieux B, Tremblay JP. Human myoblast transplantation: Preliminary results of four cases. Muscle Nerve 1992; 15: 550-60
• 27 Yao SN, Kurachi K. Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc Natl Acad Sci USA 1992; 89: 3357-61
• 28 Barr E, Leiden JM. Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 1991; 254: 1507-9
• 29 Dhawan J, Pan LC, Pavlath GK, Travis MA, Lanctot AM, Blau HM. Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 1991; 254: 1509-12
• 30 Dai Y, Roman M, Naviaux RK, Verma IM. Gene therapy via primary myoblasts: Long-term expression of factor IX protein following transplantation in vivo . Proc Natl Acad Sci USA 1992; 89: 10892-5
• 31 Yao SN, Kurachi K. Implanted myoblasts not only fuse with existing myofibers, but also survive as muscle precursor cells. Submitted for publication.