Gene therapy for haemophilia

British Journal of Haematology

Volumn 115, Issue 4, 2001, Pages 744-757

  Pasi, K John ^a  

^a UNIVERSITY OF LEICESTER   (United Kingdom)

Author keywords

Factor IX; Factor VIII; Gene therapy; Haemophilia

Indexed keywords

RECOMBINANT DNA; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; CLINICAL TRIAL; DISEASE MODEL; GENE TARGETING; GENE THERAPY; HEMOPHILIA A; HEMOPHILIA B; HUMAN; IN VIVO STUDY; LENTIVIRINAE; NONHUMAN; PATIENT SELECTION; PRIORITY JOURNAL; RETROVIRUS; REVIEW;

ADENOVIRIDAE; CLINICAL TRIALS, PHASE I; FACTOR IX; FACTOR VIII; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA A; HEMOPHILIA B; HUMANS; RETROVIRIDAE;

EID: 0035676918     PISSN: 00071048     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1365-2141.2001.03225.x     Document Type: Review

References (115)

1. Human gene therapy
2. Expression of human factor IX in rabbit hepatocytes by retrovirus mediated gene transfer: Potential for gene therapy of hemophilia B
3. Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilic dogs
4. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
5. Second generation, B-domain deleted recombinant factor VIII
6. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
7. Human germline gene modification: A dissent
8. Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes
9. Adenoviral vector-mediated expression of physiologic levels of human factor VIII in nonhuman primates
10. In vivo dose threshold effect of adenovirus-mediated factor VIII gene therapy in hemophiliac mice
11. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells
12. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
13. Persistent expression of canine factor IX in hemophilia B canines
14. Sustained expression of human factor VIII in mice using a parvovirus-based vector
15. Persistence in muscle of an adenoviral vector that lacks all viral genes
16. Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A
17. Bone marrow stromal cells as targets for gene therapy of hemophilia A
18. Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice
19. In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice
20. Sustained expression of therapeutic levels of human factor VIII in mice
21. High-level tissue-specific expression of functional human factor VIII in mice
22. Complete short-term correction of canine hemophilia A by in vivo gene therapy
23. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
24. Evaluation of an adenoviral vector encoding full-length human factor VIII in hemophiliac mice
25. Gene therapy via primary myoblasts: Long-term expression of factor IX protein following transplantation in vivo
26. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression
27. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer
28. Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice
29. Prions and haemophilia: Assessment of risk
30. The human clotting factor VIII cDNA contains an autonomously replicating sequence consensus- and matrix attachment region-like sequence that binds a nuclear factor, represses heterologous gene expression, and mediates the transcriptional effects of sodium butyrate
31. Gene therapy for haemophilia B: Host immunosuppression prolongs the therapeutic effect of adenovirus mediated gene transfer
32. Lack of persistence of E1- recombinant adenoviral vectors containing a temperature-sensitive E2A mutation in immunocompetent mice and hemophilia B dogs
33. Pre-clinical efficacy and safety studies of a gutless adenovirus vector (MAXADFVIII) for treatment of hemophilia A
34. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
35. Recombinant adeno-associated virus for muscle directed gene therapy
36. In vivo evaluation of an adenoviral vector encoding canine factor VIII: High-level, sustained expression in hemophiliac mice
37. Towards gene therapy for haemophilia B using primary human keratinocytes
38. Recombinant factor IX secreted by transduced human keratinocytes is biologically active
39. Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: Applicability to haemophilia A gene therapy
40. Animal testing of retroviral-mediated gene therapy for factor VIII deficiency
41. Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter
42. Expression of biologically active human factor IX in human hematopoietic cells after retroviral vector-mediated gene transduction
43. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
44. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
45. Expression of functional factor VIII in primary human skin fibroblasts after retrovirus mediated transfer
46. Inactivation of the Moloney murine leukemia virus long terminal repeat in murine fibroblast cell lines is associated with methylation and dependent on its chromosomal position
47. Toward gene therapy for haemophilia A: Retrovirus mediated transfer of a factor VIII gene into murine haemopoietic progenitor cells
48. Toward gene therapy for haemophilia A: Long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice
49. Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region
50. Retroviral mediated transfer and amplification of a function human factor VIII gene
51. Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells
52. In vivo gene therapy of haemophilia B: Sustained partial correction in factor IX-deficient dogs
53. In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in haemophilia B dogs
54. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
55. Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation
56. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors
57. Site-specific integration by adeno-associated virus
58. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides
59. Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide
60. Development of cell lines capable of complementing E1, E4, and protein IX defective adenovirus type 5 mutants
61. Human factor IX corrects the bleeding diathesis of mice with hemophilia B
62. Passage through mitosis is required for oncoretroviruses but not for the human immuno-deficiency virus
63. Extravascular administration of factor IX: Potential for replacement therapy of canine and human hemophilia B
64. A coagulation factor IX-deficient mouse model for human hemophilia B
65. Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero
66. Gut epithelial cells as targets for gene therapy of hemophilia
67. Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity
68. Stage-1 clinical trial of gene therapy for haemophilia B
69. Chromosomal transposition of a Tc1/mariner-like element in mouse embryonic stem cells
70. Toward somatic cell gene therapy using endothelial cells
71. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production
72. Anti-vector immunoglobulin induced by retroviral vectors
73. A phase I trial of AAV-mediated muscle directed gene transfer for hemophilia B
74. A phase I trial of AAV-mediated muscle directed gene transfer for hemophilia B
75. Adeno-associated virus vectors can be efficiently produced without helper virus
76. 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
77. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
78. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene
79. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver
80. In vivo gene delivery by lentiral vectors
81. Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA
82. An ex vivo keratinocyte model for gene therapy of hemophilia B
83. Production of human factor IX in animals by genetically modified skin fibroblasts. Potential therapy for hemophilia B
84. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver
85. Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice
86. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells
87. Phase I trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous injection
88. Non-viral gene transfer of blood coagulation factor VIII in patients with severe hemophilia A
89. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
90. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII
91. Therapeutic plasma concentrations of human factor IX in mice after gene delivery into the amniotic cavity: A model for the prenatal treatment of haemophilia B
92. Transient immunosuppression permits successful repetitive intravenous administration of an adenovirus vector
93. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
94. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
95. An alternative approach to somatic cell gene therapy
96. Sensitization of cells and retroviruses to human serum by (alpha 1-3) galactosyltransferase
97. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
98. Successful expression of human factor IX following repeat administration of adenoviral vector in mice
99. A factor IX-deficient mouse model for hemophilia B gene therapy
100. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
101. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
102. Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA
103. Long-term expression of human clotting factor IX from retrovirally transduced primary human keratinocytes in vivo
104. A new variant of Creutzfeldt-Jakob disease in the UK
105. Long term persistence of plasmid DNA and foreign gene expression in the mouse muscle
106. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
107. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
108. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy
109. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis
110. Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system
111. Expression of human factor IX in mice after injection of genetically modified myoblasts
112. Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B
113. Primary myoblast-mediated gene transfer: Persistent expression of human factor IX in mice
114. In vivo production of human factor VIII in mice after intrasplenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene delivery
115. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA