Mögliche Techniken für eine Hämophilie-Gentherapie

作者: R. Schwaab , J.-P. Faber , J. Oldenburg , H.-H. Brackmann

DOI: 10.1007/978-3-642-79009-6_20

关键词:

摘要: Die Hamophilie wird durch teilweise funktionsunfahige oder nicht vorhandene Gerinnungsproteine ausgelost. Ist das Faktor VIII-Protein defekt, liegt eine A vor. dagegen IX-Protein spricht man von einer B. Beide Erbkrankheiten werden Mutationen im VIII- bzw. IX-Gen ausgelost [1, 2].

参考文章(14)
Randal J. Kaufman, Expression and Structure-Function Properties of Recombinant Factor VIII Transfusion Medicine Reviews. ,vol. 6, pp. 235- 246 ,(1992) , 10.1016/S0887-7963(92)70174-6
Steven Limentani, David Roth, Barbara Furie, Bruce Furie, Recombinant blood clotting proteins for hemophilia therapy Seminars in Thrombosis and Hemostasis. ,vol. 19, pp. 62- 72 ,(1993) , 10.1055/S-2007-994007
Karen F. Kozarsky, James M. Wilson, Gene therapy: adenovirus vectors. Current Opinion in Genetics & Development. ,vol. 3, pp. 499- 503 ,(1993) , 10.1016/0959-437X(93)90126-A
Kenneth M. Brinkhous, Gene transfer in the hemophilias: Retrospect and prospect Thrombosis Research. ,vol. 67, pp. 329- 338 ,(1992) , 10.1016/0049-3848(92)90008-X
T Friedmann, Progress toward human gene therapy Science. ,vol. 244, pp. 1275- 1281 ,(1989) , 10.1126/SCIENCE.2660259
A. Dusty Miller, Retrovirus packaging cells. Human Gene Therapy. ,vol. 1, pp. 5- 14 ,(1990) , 10.1089/HUM.1990.1.1-5
R. Mulligan, The basic science of gene therapy Science. ,vol. 260, pp. 926- 932 ,(1993) , 10.1126/SCIENCE.8493530
E.G.D. Tuddenham, D.N. Cooper, J. Gitschier, M. Higuchi, L.W. Hoyer, A. Yoshioka, I.R. Peake, R. Schwaab, K. Olek, H.H. Kazazian, J.M. Lavergne, F. Giannelli, S.E. Antonarakis, Haemophilia A: database of ncleotide substituttions, deletions, insertions and rearrangements of the factor VIII gene Nucleic Acids Research. ,vol. 19, pp. 4821- 4833 ,(1991) , 10.1093/NAR/19.18.4821
Claudio Bordignon, Fulvio Mavilio, Giuliana Ferrari, Paolo Servida, Alberto G. Ugazio, Luigi D. Notarangelo, Eli Gilboa, Silvano Rossini, Richard J. O'Reilly, Clayton A. Smith, Alfred P. Gillio, W. French Anderson, R. Michael Blaese, Robert C. Moen, Martin A. Eglitis, Transfer of the ADA Gene into Bone Marrow Cells and Peripheral Blood Lymphocytes for the Treatment of Patients Affected by ADA-Deficient SCID Human Gene Therapy. ,vol. 4, pp. 513- 520 ,(1993) , 10.1089/HUM.1993.4.4-513
Joseph Zabner, Larry A. Couture, Richard J. Gregory, Scott M. Graham, Alan E. Smith, Michael J. Welsh, Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis Cell. ,vol. 75, pp. 207- 216 ,(1993) , 10.1016/0092-8674(93)80063-K