Gene Therapy for Hemophilia B With Liver-specific Element Mediated by Rep-RBE Site-specific Integration System
暂无分享,去创建一个
Juan Ye | Jin-zhong Chen | Jing-lun Xue | Q. Shen | Zhengxin Xu | Amin Zhang | Linjun Xie
[1] Hong-Sung Kim,et al. Hepatic control elements promote long‐term expression of human coagulation factor IX gene in hydrodynamically transfected mice , 2011, The journal of gene medicine.
[2] K. High,et al. Gene therapy for haemophilia: a long and winding road , 2011, Journal of thrombosis and haemostasis : JTH.
[3] T. VandenDriessche,et al. Gene therapy strategies for hemophilia: benefits versus risks , 2010, The journal of gene medicine.
[4] E. Geissler,et al. High volume naked DNA tail‐vein injection restores liver function in Fah‐knock out mice , 2010, Journal of gastroenterology and hepatology.
[5] W. Jia,et al. A 16-bp RBE element mediated Rep-dependent site-specific integration in AAVS1 transgenic mice for expression of hFIX , 2009, Gene Therapy.
[6] Peter Mayrhofer,et al. Use of minicircle plasmids for gene therapy. , 2009, Methods in molecular biology.
[7] M. Sadelain,et al. Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[8] K. L. Douglas. Toward Development of Artificial Viruses for Gene Therapy: A Comparative Evaluation of Viral and Non‐viral Transfection , 2008, Biotechnology progress.
[9] B. Geest,et al. Direct comparison of hepatocyte-specific expression cassettes following adenoviral and nonviral hydrodynamic gene transfer , 2008, Gene Therapy.
[10] W. Jia,et al. A 16bp Rep binding element is sufficient for mediating Rep-dependent integration into AAVS1. , 2006, Journal of molecular biology.
[11] Geoffrey Kemball-Cook,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. , 2006, Blood.
[12] T. Flotte,et al. DNA-dependent PK inhibits adeno-associated virus DNA integration , 2004, Proceedings of the National Academy of Sciences of the United States of America.
[13] Theresa A. Storm,et al. Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo , 2001, Journal of Virology.
[14] D. Treco,et al. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. , 2001, The New England journal of medicine.
[15] K. Loeb,et al. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.
[16] I. Peake. Gene therapy for haemophilia? , 2001 .
[17] Guisheng Zhang,et al. Long-term expression of human alpha1-antitrypsin gene in mouse liver achieved by intravenous administration of plasmid DNA using a hydrodynamics-based procedure , 2000, Gene Therapy.
[18] M. Kay,et al. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.
[19] J. Wolff,et al. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. , 1999, Human gene therapy.
[20] Dexi Liu,et al. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA , 1999, Gene Therapy.
[21] R. Kotin,et al. Determination of adeno-associated virus Rep68 and Rep78 binding sites by random sequence oligonucleotide selection , 1995, Journal of virology.
[22] M. W. Flye,et al. Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes. , 1994, Blood.
[23] M. Kay,et al. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. , 1993, Science.
[24] R. Kotin,et al. Characterization of a preferred site on human chromosome 19q for integration of adeno‐associated virus DNA by non‐homologous recombination. , 1992, The EMBO journal.
[25] P. Leder,et al. The cytomegalovirus enhancer: a pan-active control element in transgenic mice , 1990, Molecular and cellular biology.