SEARCH:   Advanced

Blood, 15 February 2004, Vol. 103, No. 4, pp. 1253-1260. Prepublished online as a Blood First Edition Paper on October 9, 2003; DOI 10.1182/blood-2003-08-2954. This Article Full Text (PDF) All Versions of this Article: 2003-08-2954v1 103/4/1253    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Sarkar, R. Articles by Kazazian, H. H Search for Related Content PubMed PubMed Citation Articles by Sarkar, R. Articles by Kazazian, H. H Related Collections Related Article in Blood Online Social Bookmarking                   What's this? Submitted August 29, 2003 Accepted September 26, 2003 Total correction of hemophilia A mice with canine FVIII using an AAV8 serotype Rita Sarkar*, Renee Tetreault, Guangping Gao, Lili Wang, Peter Bell, Randy Chandler, James M Wilson, and Haig H Kazazian Department of Genetics, University of Pennsylvania, Philadelphia, PA, USA Department of Medical Genetics, University of Pennsylvania, Philadelphia, PA, USA * Corresponding author; email: sarkarr{at}mail.med.upenn.edu. Despite the popularity of AAV2 (adeno-associated virus 2) as a vehicle for gene transfer, its efficacy for liver-directed gene therapy in hemophilia A or B has been suboptimal. Here we evaluated AAV serotypes 2, 5, 7 and 8 in gene therapy of factor VIII deficiency in a hemophilia A mouse model and found that AAV8 was superior to the other three serotypes. We expressed canine B-domain deleted FVIII cDNA either in a single vector or in two separate AAV vectors containing the heavy and light chain cDNAs. We also evaluated AAV8 against AAV2 in intraportal and tail vein injections. AAV8 gave 100% correction of plasma FVIII activity irrespective of the vector type or route of administration. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Article in Blood Online: Swinging for the fences: persistent and efficient liver-directed gene therapy for hemophilia A Terence R. Flotte Blood 2004 103: 1183-1184. [Full Text] [PDF] This article has been cited by other articles: D. E. Sabatino, C. F. Freguia, R. Toso, A. Santos, E. P. Merricks, H. H. Kazazian Jr, T. C. Nichols, R. M. Camire, and V. R. Arruda Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model Blood, November 12, 2009; 114(20): 4562 - 4565. [Abstract] [Full Text] [PDF] K. J. Ho, C. E. Bass, A. H. K. Kroemer, C. Ma, E. Terwilliger, and S. J. Karp Optimized adeno-associated virus 8 produces hepatocyte-specific Cre-mediated recombination without toxicity or affecting liver regeneration Am J Physiol Gastrointest Liver Physiol, August 1, 2008; 295(2): G412 - G419. [Abstract] [Full Text] [PDF] H.-J. Nam, M. D. Lane, E. Padron, B. Gurda, R. McKenna, E. Kohlbrenner, G. Aslanidi, B. Byrne, N. Muzyczka, S. Zolotukhin, et al. Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector J. Virol., November 15, 2007; 81(22): 12260 - 12271. [Abstract] [Full Text] [PDF] H. Jiang, L. B. Couto, S. Patarroyo-White, T. Liu, D. Nagy, J. A. Vargas, S. Zhou, C. D. Scallan, J. Sommer, S. Vijay, et al. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy Blood, November 15, 2006; 108(10): 3321 - 3328. [Abstract] [Full Text] [PDF] K. Kitajima, D. H.L. Marchadier, G. C. Miller, G.-p. Gao, J. M. Wilson, and D. J. Rader Complete Prevention of Atherosclerosis in ApoE-Deficient Mice by Hepatic Human ApoE Gene Transfer With Adeno-Associated Virus Serotypes 7 and 8 Arterioscler Thromb Vasc Biol, August 1, 2006; 26(8): 1852 - 1857. [Abstract] [Full Text] [PDF] H. Jiang, D. Lillicrap, S. Patarroyo-White, T. Liu, X. Qian, C. D. Scallan, S. Powell, T. Keller, M. McMurray, A. Labelle, et al. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs Blood, July 1, 2006; 108(1): 107 - 115. [Abstract] [Full Text] [PDF] D. Grimm, K. Pandey, H. Nakai, T. A. Storm, and M. A. Kay Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype J. Virol., January 1, 2006; 80(1): 426 - 439. [Abstract] [Full Text] [PDF] X. Liu, Z. Yan, M. Luo, and J. F. Engelhardt Species-Specific Differences in Mouse and Human Airway Epithelial Biology of Recombinant Adeno-Associated Virus Transduction Am. J. Respir. Cell Mol. Biol., January 1, 2006; 34(1): 56 - 64. [Abstract] [Full Text] [PDF] Y. Kang, L. Xie, D. T. Tran, C. S. Stein, M. Hickey, B. L. Davidson, and P. B. McCray Jr Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer Blood, September 1, 2005; 106(5): 1552 - 1558. [Abstract] [Full Text] [PDF] J. C. Grieger and R. J. Samulski Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps J. Virol., August 1, 2005; 79(15): 9933 - 9944. [Abstract] [Full Text] [PDF] L. Xu, T. C. Nichols, R. Sarkar, S. McCorquodale, D. A. Bellinger, and K. P. Ponder Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy PNAS, April 26, 2005; 102(17): 6080 - 6085. [Abstract] [Full Text] [PDF] L. Wang, R. Calcedo, T. C. Nichols, D. A. Bellinger, A. Dillow, I. M. Verma, and J. M. Wilson Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy Blood, April 15, 2005; 105(8): 3079 - 3086. [Abstract] [Full Text] [PDF] H. Nakai, S. Fuess, T. A. Storm, S.-i. Muramatsu, Y. Nara, and M. A. Kay Unrestricted Hepatocyte Transduction with Adeno-Associated Virus Serotype 8 Vectors in Mice J. Virol., January 1, 2005; 79(1): 214 - 224. [Abstract] [Full Text] [PDF] Z. Sandalon, E. M. Bruckheimer, K. H. Lustig, L. C. Rogers, R. W. Peluso, and H. Burstein Secretion of a TNFR:Fc Fusion Protein following Pulmonary Administration of Pseudotyped Adeno-Associated Virus Vectors J. Virol., November 15, 2004; 78(22): 12355 - 12365. [Abstract] [Full Text] [PDF] E. Dobrzynski, F. Mingozzi, Y.-L. Liu, E. Bendo, O. Cao, L. Wang, and R. W. Herzog Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer Blood, August 15, 2004; 104(4): 969 - 977. [Abstract] [Full Text] [PDF]

	Copyright © 2003 by American Society of Hematology         Online ISSN: 1528-0020