Progress in the molecular biology of inherited bleeding disorders
dc.contributor.author | Pipe, Steven W. | en_US |
dc.contributor.author | High, K. A. | en_US |
dc.contributor.author | Ohashi, K. | en_US |
dc.contributor.author | Ural, A. U. | en_US |
dc.contributor.author | Lillicrap, D. | en_US |
dc.date.accessioned | 2010-06-01T20:16:18Z | |
dc.date.available | 2010-06-01T20:16:18Z | |
dc.date.issued | 2008-07 | en_US |
dc.identifier.citation | PIPE, S. W.; HIGH, K. A.; OHASHI, K.; URAL, A. U.; LILLICRAP, D. (2008). "Progress in the molecular biology of inherited bleeding disorders." Haemophilia 14(s3 State of the Art. XXVIII International Congress of the World Federation of Hemophilia ): 130-137. <http://hdl.handle.net/2027.42/73391> | en_US |
dc.identifier.issn | 1351-8216 | en_US |
dc.identifier.issn | 1365-2516 | en_US |
dc.identifier.uri | https://hdl.handle.net/2027.42/73391 | |
dc.identifier.uri | http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=retrieve&db=pubmed&list_uids=18510533&dopt=citation | en_US |
dc.format.extent | 143744 bytes | |
dc.format.extent | 3109 bytes | |
dc.format.mimetype | application/pdf | |
dc.format.mimetype | text/plain | |
dc.publisher | Blackwell Publishing Ltd | en_US |
dc.rights | © 2008 Blackwell Publishing Ltd | en_US |
dc.title | Progress in the molecular biology of inherited bleeding disorders | en_US |
dc.type | Article | en_US |
dc.subject.hlbsecondlevel | Oncology and Hematology | en_US |
dc.subject.hlbtoplevel | Health Sciences | en_US |
dc.description.peerreviewed | Peer Reviewed | en_US |
dc.contributor.affiliationum | * Department of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, USA | en_US |
dc.contributor.affiliationother | † Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA | en_US |
dc.contributor.affiliationother | † Institute of Advanced Biomedical Engineering and Science and Department of Surgery, Tokyo Women’s Medical University, Tokyo, Japan | en_US |
dc.contributor.affiliationother | § Department of Haematology, Gulhane Military Medical Faculty, Medical and Cancer Research Center, Etlik-Ankara, Turkey | en_US |
dc.contributor.affiliationother | ¶ Department of Pathology & Molecular Medicine, Queen’s University, Kingston, Canada | en_US |
dc.identifier.pmid | 18510533 | en_US |
dc.description.bitstreamurl | http://deepblue.lib.umich.edu/bitstream/2027.42/73391/1/j.1365-2516.2008.01718.x.pdf | |
dc.identifier.doi | 10.1111/j.1365-2516.2008.01718.x | en_US |
dc.identifier.source | Haemophilia | en_US |
dc.identifier.citedreference | Pipe SW. The promise and challenges of bioengineered recombinant clotting factors. J Thromb Haemost 2005; 3: 1692 – 701. | en_US |
dc.identifier.citedreference | Saenko EL, Pipe SW. Strategies towards a longer acting factor VIII. Haemophilia 2006; 12 ( Suppl 3 ): 42 – 51. | en_US |
dc.identifier.citedreference | Barrow RT, Lollar P. Neutralization of antifactor VIII inhibitors by recombinant porcine factor VIII. J Thromb Haemost 2006; 4: 2223 – 9. | en_US |
dc.identifier.citedreference | Bitonti AJ, Dumont JA, Low SC et al. Pulmonary delivery of an erythropoietin Fc fusion protein in non-human primates through an immunoglobulin transport pathway. Proc Natl Acad Sci USA 2004; 101: 9763 – 8. | en_US |
dc.identifier.citedreference | Tranholm M, Kristensen K, Kristensen AT, Pyke C, Rojkjaer R, Persson E. Improved hemostasis with superactive analogs of factor VIIa in a mouse model of hemophilia A. Blood 2003; 102: 3615 – 20. | en_US |
dc.identifier.citedreference | Margaritis P, High KA. Advances in gene therapy using factor VIIa in hemophilia. Semin Hematol 2006; 43: S101 – 4. | en_US |
dc.identifier.citedreference | Pollak E, High KA. Genetic disorders of coagulation. In: Warrell D, Cox T, Firth J, Benz E, eds. Oxford Textbook of Medicine, Vol. 3, 4th edn. Oxford: Oxford University Press, 2003: 757 – 67. | en_US |
dc.identifier.citedreference | Schwarzwaelder K, Howe SJ, Schmidt M et al. Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest 2007; 117: 2241 – 9. | en_US |
dc.identifier.citedreference | Powell JS, Ragni MV, White GC II et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 2003; 102: 2038 – 45. | en_US |
dc.identifier.citedreference | Roth DA, Tawa NE Jr, O’Brien JM, Treco DA, Selden RF. Factor Transkaryotic Therapy Study Group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1735 – 42. | en_US |
dc.identifier.citedreference | BalaguÉ C, Zhou J, Dai Y et al. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood 2000; 95: 820 – 8. | en_US |
dc.identifier.citedreference | Mount JD, Herzog RW, Tillson DM et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 2002; 99: 2670 – 6. | en_US |
dc.identifier.citedreference | Donsante A, Vogler C, Muzyczka N et al. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther 2001; 8: 1343 – 6. | en_US |
dc.identifier.citedreference | Donsante A, Miller DG, Li Y et al. AAV vector integration sites in mouse hepatocellular carcinoma. Science 2007; 317: 477. | en_US |
dc.identifier.citedreference | Manno CS, Chew AJ, Hutchison S et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003; 101: 2963 – 72. | en_US |
dc.identifier.citedreference | Arruda VR, Stedman HH, Nichols TC et al. Regional intravascular delivery of AAV-2-FIX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood 2005; 105: 3458 – 64. | en_US |
dc.identifier.citedreference | Wang L, Calcedo R, Nichols TC et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 2005; 105: 3079 – 86. | en_US |
dc.identifier.citedreference | Scallan CD, Lillicrap D, Jiang H et al. Sustained phenotypic correction of canine hemophilia A using an adeno-associate viral vector. Blood 2003; 102: 2031 – 7. | en_US |
dc.identifier.citedreference | Manno CS, Pierce GF, Arruda VR et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342 – 7. | en_US |
dc.identifier.citedreference | Mingozzi F, Maus MV, Hui DJ et al. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 2007; 13: 419 – 22. | en_US |
dc.identifier.citedreference | Nathwani AC, Gray JT, Ng CY et al. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 2006; 107: 2653 – 61. | en_US |
dc.identifier.citedreference | Dhawan A, Mitry RR, Hughes RD et al. Hepatocyte transplantation for inherited factor VII deficiency. Transplantation 2004; 78: 1812 – 4. | en_US |
dc.identifier.citedreference | Yokoyama T, Ohashi K, Kuge H et al. In vivo engineering of metabolically active hepatic tissues in a neovascularized subcutaneous cavity. Am J Transplant 2006; 6: 50 – 9. | en_US |
dc.identifier.citedreference | Ohashi K, Waugh JM, Dake MD et al. Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases. Hepatology 2005; 41: 132 – 40. | en_US |
dc.identifier.citedreference | Ohashi K, Marion PL, Nakai H et al. Sustained survival of human hepatocytes in mice: a model for in vivo infection with human hepatitis B and hepatitis delta viruses. Nat Med 2000; 6: 327. | en_US |
dc.identifier.citedreference | Ohashi K. Liver tissue engineering: the future of liver therapeutics. Hepatol Res 2008 ( in press ). | en_US |
dc.identifier.citedreference | Ohashi K, Yokoyama T, Yamato M et al. Engineering functional two- and three-dimensional liver systems in vivo using hepatic tissue sheets. Nat Med 2007; 13: 880 – 5. | en_US |
dc.identifier.citedreference | Yang J, Yamato M, Shimizu T et al. Reconstruction of functional tissues with cell sheet engineering. Biomaterials 2007; 28: 5033 – 43. | en_US |
dc.identifier.citedreference | Oh T, Peister A, Ohashi K, Park F. Transplantation of murine bone marrow stromal cells under the kidney capsule to secrete coagulation factor VIII. Cell Transplant 2006; 15: 637 – 45. | en_US |
dc.identifier.citedreference | Roosendaal G, Lafeber FP. Pathogenesis of haemophilic arthropathy. Haemophilia 2006; 12 ( Suppl 3 ): 117 – 21. | en_US |
dc.identifier.citedreference | Hoots WK. Pathogenesis of hemophilic arthropathy. Semin Hematol 2006; 43 ( Suppl 1 ): S18 – 22. | en_US |
dc.identifier.citedreference | De Bari C, Dell’accio F. Mesenchymal stem cells in rheumatology: a regenerative approach to joint repair. Clin Sci 2007; 113: 339 – 48. | en_US |
dc.identifier.citedreference | Lukic IK, Grcevic D, Kovacic N et al. Alteration of newly induced endochondral bone formation in adult mice without tumour necrosis factor receptor 1. Clin Exp Immunol 2005; 139: 236 – 44. | en_US |
dc.identifier.citedreference | Shealy DJ, Wooley PH, Emmell E et al. Anti-TNF-alpha antibody allows healing of joint damage in polyarthritic transgenic mice. Arhritis Res 2002; 4: R7. | en_US |
dc.owningcollname | Interdisciplinary and Peer-Reviewed |
Files in this item
Remediation of Harmful Language
The University of Michigan Library aims to describe library materials in a way that respects the people and communities who create, use, and are represented in our collections. Report harmful or offensive language in catalog records, finding aids, or elsewhere in our collections anonymously through our metadata feedback form. More information at Remediation of Harmful Language.
Accessibility
If you are unable to use this file in its current format, please select the Contact Us link and we can modify it to make it more accessible to you.