Status of Gene Transfer for Hemophilia A and B

• References

• 1 Miller AD. Progress toward human gene therapy. Blood 1990; 76: 271-78
• 2 Bontempo FA, Lewis JH, Gorenc TJ, Spero JA, Ragni MV, Scott JP, Starzl TE. Liver transplantation in hemophilia A. Blood 1987; 69: 1721-24
• 3 St Louis D, Verma IM. An alternative approach to somatic cell gene therapy. Proc. of Natl. Acad. Sci. USA 1988; 85: 3150-54
• 4 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
• 5 Axelrod JH, Read MS, Brinkhous KM, Verma IM. Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilic dogs. Proc. of Natl. Acad. Sci. USA 1990; 87: 5173-77
• 6 Palmer TD, Rosman GJ, Osborne WRA, Miller AD. Genetically-modified skin fibroblasts persist long-term after transplantation but gradually inactivate introduced genes. Proc. of Natl. Acad. Sci. USA 1991; 80: 1330-34
• 7 Lynch CM, Israel DI, Miller AD. Towards somatic cell gene therapy using endothelial cells. J. of Cell Biochem. (Suppl) 1990; 14E: 22
• 8 Israel DI, Kaufman RJ. Retroviral-mediated transfer and amplification of a functional human factor VIII gene. Blood 1990; 75: 1074-80
• 9 Hoeben RC, van der Jagt RCM, Schoute F, van Tilburg NH, Verbeet MPh, Briet E, van Ormondt H, van der Eb AJ. Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer. J. of Biol. Chem. 1990; 265: 7318-7323
• 10 Armentano D, Thompson AR, Darlington G, Woo SLC. Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: Potential for gene therapy of hemophilia B. Proc. of Natl. Acad. Sci. USA 1990; 87: 6141-45
• 11 Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, Lotze MT, Yang JC, Topalian SL, Merino MJ, Culver K, Miller AD, Blaese RM, Anderson WF. Gene transfer into humans: Immunotherapy of patients with advanced melanoma using tumor infiltrating lymphocytes modified by retroviral gene transduction. N. Eng. J. Med. 1990; 323: 57
• 12 Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, Jani A, Feigner PL. Direct gene transfer into mouse muscle in vivo. . Science 1990; 247: 1465
• 13 Bodine DM, McDonagh KT, Brandt SJ, Ney PA, Agricola B, Byrne E, Nienhuis AW. Development of a high titer retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells. Proc. of Natl. Acad. Sci. USA 1990; 87: 373
• 14 Pittman DD, Kaufman RJ. Structure-function relationship of factor VIII elucidated through recombinant DNA technology. Thromb. Haemost. 1989; 61: 161-65
• 15 Smith KJ, Thompson AR. Labelled factor IX kinetics in patients with hemophilia B. Blood 1981; 58: 625-29