PDF herunterladen
Thromb Haemost 2002; 87(03): 362-363
DOI: 10.1055/s-0037-1613011
Commentary
Schattauer GmbH

Platelets: A New Target for Gene Therapy of Haemophilia B

Mark A. Brown
1   Department of Clinical Chemistry, Lund University, Malmö, Sweden
› Institutsangaben
Weitere Informationen

Publikationsverlauf

Publikationsdatum:
14. Dezember 2017 (online)

Auch verfügbar auf
    Lizenzen und Reprints

 

 

  • References

  • 1 St Louis D, Verma IM. An alternative approach to somatic cell gene therapy. Proc Natl Acad Sci USA 1988; 85: 3150-54.
    CrossrefPubMedSuche in Google Scholar
  • 2 Giannelli F, Green PM, Sommer SS, Poon M-C, Ludwig M, Schwaab R, Reitsma PH, Goossens M, Yoshioka A, Figueiredo MS, Brownlee GG. Haemophilia B: database of point mutations and short additions and deletions – eighth edition. Nucleic Acids Res 1998; 26: 265-8.
    CrossrefPubMedSuche in Google Scholar
  • 3 Sommer SS, Scaringe WA, Hill KA. Human germline mutation in the factor IX gene. Mutat Res 2001; 487: 1-17.
    CrossrefPubMedSuche in Google Scholar
  • 4 Aledort LM. Economic aspects of haemophilia care in the United States. Haemophilia 1999; 05: 282-5.
    CrossrefPubMedSuche in Google Scholar
  • 5 Kaufman RJ. Advances toward gene therapy for hemophilia at the millennium. Hum Gene Ther 1999; 10: 2091-107.
    CrossrefPubMedSuche in Google Scholar
  • 6 Chuah MK, Collen D, VandenDriessche T. Gene therapy for hemophilia. J Gene Med 2001; 03: 3-20.
    CrossrefPubMedSuche in Google Scholar
  • 7 Qiu X, Lu D, Zhou J, Wang J, Yang J, Meng P, Hsueh JL. Implantation of autologous skin fibroblasts genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients. Chin Med J 1996; 109: 832-9.
    PubMedSuche in Google Scholar
  • 8 Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 2000; 24: 257-61.
    CrossrefPubMedSuche in Google Scholar
  • 9 Cornetta K. Safety aspects of gene therapy. Br J Haematol 1992; 80: 421-6.
    CrossrefPubMedSuche in Google Scholar
  • 10 High KA. Gene transfer as an approach to treating hemophilia. Circ Res 2001; 88: 137-44.
    CrossrefPubMedSuche in Google Scholar
  • 11 Vander Velden P, Giles AR. A detailed morphological evaluation of the evolution of the haemostatic plug in normal, factor VII and factor VIII deficient dogs. Br J Haematol 1988; 70: 345-55.
    CrossrefPubMedSuche in Google Scholar
  • 12 Giesen PL, Rauch U, Bohrmann B, Kling D, Roqué M, Fallon JT, Badimon JJ, Himber J, Riederer MA, Nemerson Y. Blood-borne tissue factor: another view of thrombosis. Proc Natl Acad Sci USA 1999; 96: 2311-5.
    CrossrefPubMedSuche in Google Scholar
  • 13 Gailani D, Ho D, Sun MF, Cheng Q, Walsh PN. Model for a factor IX activation complex on blood platelets: dimeric conformation of factor XIa is essential. Blood 2001; 97: 3117-22.
    CrossrefPubMedSuche in Google Scholar
  • 14 Majerus PW. Platelets. In: Stamatoyannopoulos G, Majerus PW, Perlmutter RM, Varmus H. eds. The Molecular Basis of Blood Diseases. Philadelphia: WB Saunders Co; 2001: 764-90.
    Suche in Google Scholar
  • 15 Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients – a long-term follow-up. J Intern Med 1997; 241: 395-400.
    CrossrefPubMedSuche in Google Scholar
  • 16 Wilcox DA, Olsen JC, Ishizawa L, Griffith M, White GCI. Integrin αIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells. Proc Natl Acad Sci USA 1999; 96: 9654-9.
    CrossrefPubMedSuche in Google Scholar
  • 17 Stenflo J, Dahlbäck B. Vitamin K-dependent proteins in blood coagulation. In: Stamatoyannopoulos G, Majerus PW, Perlmutter RM, Varmus H. eds. The Molecular Basis of Blood Diseases. Philadelphia: WB Saunders Co; 2001: 579-613.
    Suche in Google Scholar
  • 18 Hao Q-L, Malik P, Salazar R, Tang H, Gordon EM, Kohn DB. Expression of biologically active human factor IX in human hematopoietic cells after retroviral vector-mediated gene transduction. Hum Gene Ther 1995; 06: 873-80.
    CrossrefPubMedSuche in Google Scholar
  • 19 Arruda VR, Hagstrom JN, Deitch J, Heiman-Patterson T, Camire RM, Chu K, Fields PA, Herzog RW, Couto LB, Larson PJ, High KA. Posttranslational modifications of recombinant myotube-synthesized human factor IX. Blood 2001; 97: 130-8.
    CrossrefPubMedSuche in Google Scholar
  • 20 Plantier J-L, Enjolras N, Rodriguez M-H, Négrier C. P-selectin tail targets the factor IX in secretory granules of AtT20 cells. Thromb Haemost 1999; 82 Suppl Abstract 1505.
    PubMedSuche in Google Scholar
  • 21 Plantier J-L, Enjolras N, Rodriguez M-H, Négrier C. P-selectin tail induces the storage of factor IX in CHO cells. Blood 1999; 92 10, Suppl 1 380b.
    PubMedSuche in Google Scholar
  • 22 Tronik-Le Roux D, Roullot V, Schweitzer A, Berthier R, Marguerie G. Suppression of erythro-megakaryocytopoiesis and the induction of reversible thrombocytopenia in mice transgenic for the thymidine kinase gene targeted by the platelet glycoprotein αIIb promoter. J Exp Med 1995; 181: 2141-51.
    CrossrefPubMedSuche in Google Scholar
  • 23 Tropel P, Roullot V, Vernet M, Poujol C, Pointu H, Nurden P, Marguerie G, Tronik-Le Roux D. A 2.7-kb portion of the 5’ flanking region of the murine glycoprotein αIIb gene is transcriptionally active in primitive hematopoietic progenitor cells. Blood 1997; 90: 2995-3004.
    PubMedSuche in Google Scholar
  • 24 Cavazzana-Calvo M, Hacein-Bey S, de Saint GBasile, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288: 669-72.
    CrossrefPubMedSuche in Google Scholar