Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
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J. Rasko | M. Kay | J. Zehnder | H. Ertl | M. Ozelo | B. Konkle | J. F. Wright | H. Nakai | J. Sommer | K. High | C. Manno | M. Ragni | L. Couto | B. Glader | A. Chew | V. Arruda | M. Razavi | D. Sabatino | F. Mingozzi | G. Pierce | K. Hoots | P. Blatt | M. Dake | R. Kaye | A. Zajko | P. Rustagi | D. Leonard | R. Lessard | M. Tigges | A. Luk | Haiyan Jiang | J. F. Wright
[1] 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.
[2] J. Rasko,et al. ERRATUM: People to watch , 2006, Nature Medicine.
[3] J. F. Wright,et al. Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.
[4] H. Stedman,et al. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. , 2005, Blood.
[5] Lili Wang,et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. , 2005, Blood.
[6] W. Xiao,et al. Intracellular Viral Processing, Not Single-Stranded DNA Accumulation, Is Crucial for Recombinant Adeno-Associated Virus Transduction , 2004, Journal of Virology.
[7] L. Couto,et al. Direct exposure of mouse spermatozoa to very high concentrations of a serotype-2 adeno-associated virus gene therapy vector fails to lead to germ cell transduction. , 2004, Human gene therapy.
[8] Victor Appay,et al. Lessons from the study of T-cell differentiation in persistent human virus infection. , 2004, Seminars in immunology.
[9] Theresa A. Storm,et al. Rapid Uncoating of Vector Genomes Is the Key toEfficient Liver Transduction with Pseudotyped Adeno-Associated VirusVectors , 2004, Journal of Virology.
[10] A. Parker,et al. Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector. , 2003, Blood.
[11] B. Boehm,et al. CD4+ and CD8+ cells in cryopreserved human PBMC maintain full functionality in cytokine ELISPOT assays. , 2003, Journal of immunological methods.
[12] Alan McClelland,et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. , 2003, Blood.
[13] Alan McClelland,et al. Quantification of adeno-associated virus particles and empty capsids by optical density measurement. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.
[14] C. Slaughter,et al. Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques. , 2002, Blood.
[15] R. Herzog,et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. , 2002, Blood.
[16] Sylvia Janetzki,et al. A panel of MHC class I restricted viral peptides for use as a quality control for vaccine trial ELISPOT assays. , 2002, Journal of immunological methods.
[17] J. Biegel,et al. Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.
[18] Jean Bennett,et al. Gene therapy restores vision in a canine model of childhood blindness , 2001, Nature Genetics.
[19] 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.
[20] Alan McClelland,et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector , 2000, Nature Genetics.
[21] I. Verma,et al. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.
[22] R. Samulski,et al. Persistent expression of canine factor IX in hemophilia B canines , 1999, Gene Therapy.
[23] Katherine A. High,et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector , 1999, Nature Medicine.
[24] M. Kay,et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors , 1999, Nature Medicine.
[25] R. Ljung. Can haemophilic arthropathy be prevented? , 1998, British journal of haematology.
[26] H. Pettersson,et al. Haemophilia prophylaxis in young patients–a long‐term follow‐up , 1997, Journal of internal medicine.
[27] S. Cai,et al. Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy. , 1997, Blood.
[28] P. Bedossa,et al. An algorithm for the grading of activity in chronic hepatitis C , 1996, Hepatology.
[29] K. Kurachi,et al. Role of Intron I in Expression of the Human Factor IX Gene (*) , 1995, The Journal of Biological Chemistry.
[30] P. Bedossa,et al. Intraobserver and Interobserver Variations in Liver Biopsy Interpretation in Patients with Chronic Hepatitis C , 1994 .