RSS-Feed abonnieren
Bitte kopieren Sie die angezeigte URL und fügen sie dann in Ihren RSS-Reader ein.
https://www.thieme-connect.de/rss/thieme/de/10.1055-s-00035024.xml
PDF herunterladen
Thromb Haemost 2002; 87(03): 362-363
DOI: 10.1055/s-0037-1613011
DOI: 10.1055/s-0037-1613011
Commentary
Platelets: A New Target for Gene Therapy of Haemophilia B
Weitere Informationen
Publikationsverlauf
Publikationsdatum:
14. Dezember 2017 (online)
-
References
- 1 St Louis D, Verma IM. An alternative approach to somatic cell gene therapy. Proc Natl Acad Sci USA 1988; 85: 3150-54.
- 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.
- 3 Sommer SS, Scaringe WA, Hill KA. Human germline mutation in the factor IX gene. Mutat Res 2001; 487: 1-17.
- 4 Aledort LM. Economic aspects of haemophilia care in the United States. Haemophilia 1999; 05: 282-5.
- 5 Kaufman RJ. Advances toward gene therapy for hemophilia at the millennium. Hum Gene Ther 1999; 10: 2091-107.
- 6 Chuah MK, Collen D, VandenDriessche T. Gene therapy for hemophilia. J Gene Med 2001; 03: 3-20.
- 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.
- 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.
- 9 Cornetta K. Safety aspects of gene therapy. Br J Haematol 1992; 80: 421-6.
- 10 High KA. Gene transfer as an approach to treating hemophilia. Circ Res 2001; 88: 137-44.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.