Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs

Blood

Volumn 97, Issue 1, 2001, Pages 107-113

  Gallo Penn, Angela M ^b   Shirley, Pamela S ^b   Andrews, Julie L ^b   Tinlin, Shawn ^b   Webster, Sandy ^b   Cameron, Cherie ^b   Hough, Christine ^b   Notley, Colleen ^b   Lillicrap, David ^b   Kaleko, Michael ^b   Connelly, Sheila ^a  

^a NOVARTIS PHARMA AG   (Switzerland)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 INHIBITOR; VIRUS VECTOR;

ANIMAL CELL; ANIMAL TISSUE; ANTIBODY TITER; ARTICLE; BLOOD CLOTTING; BLOOD TOXICITY; DOG; DOSE RESPONSE; GENE THERAPY; HEMOPHILIA A; LIVER TOXICITY; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION;

EID: 0035174331     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.V97.1.107     Document Type: Article

References (49)

1. Hemophilia: A new approach to an old disease
2. Adenoviral vectors for liver-directed gene therapy
3. Haemophilia A gene therapy
4. In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice
5. Sustained expression of therapeutic levels of human factor VIII in mice
6. High level tissue-specitic expression of functional human FVIII in mice
7. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
8. Adenoviral vector-mediated expression of physiologic levels of human factor VIII in nonhuman primates
9. Complete short-term correction of canine hemophilia A by in vivo gene therapy
10. Evaluation of an adenoviral vector encoding full-length factor VIII in hemophiliac mice
11. In vivo evaluation of an adenoviral vector encoding canine factor VIII: High-level sustained expression in hemophiliac mice
12. Canine hemophilia: Observation on the course, the clotting anomaly and the effects of blood transfusion
13. A canine model of hemophilic (factor VIII:C deficiency) bleeding
14. Hemophilic dog model for evaluating therapeutic effectiveness of plasma protein fractions
15. In vivo characterization of recombinant factor VIII in a canine model of hemophilia A (factor VIII deficiency)
16. Animal models for the study of factor VIII and von Willebrand factor
17. The role of factor VII in haemostasis: Infusion studies of factor VIIa in a canine models of factor VIII deficiency
18. The roles of von Willebrand factor and factor VIII in arterial thrombosis: Studies in canine von Willebrand disease and hemophilia A
19. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII
20. The development and characterisation of antibodies to human factor VIII in haemophilic dogs
21. The development of homologous (canine/anti-canine) antibodies in dogs with haemophilia A (factor VIII deficiency): A ten-year longitudinal study
22. The canine factorr VIII cDNA and 5′ flanking sequence
23. Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy
24. Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy
25. Adenovirus vectors for gene therapy
26. Hemophilia A
27. Coagulation assays and platelet aggregation patterns in human, baboon, and canine blood
28. Activity of coagulation and fibrinolysis parameters in animals
29. A more uniform measurement of factor VIII inhibitors
30. Influence of phospholipids on the assessment of factor VIII activity
31. Adenovirus-mediated expression of therapeutic plasma levels of human factor IX in mice
32. MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses
33. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses
34. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy
35. Gene therapy for hemophilia B: Host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression
36. The mouse
37. Transient immunosuppression by FK506 permits a sustained high-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscles of adult dystrophic (mdx) mice
38. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-detective adenovirus vectors
39. Role of viral antigens in destructive cellular immune responses to adenovirus vector transduce cells in mouse lungs
40. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains
41. High-capacity adenoviral vectors for gene transfer and somatic gene therapy
42. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene
43. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
44. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
45. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity
46. Adenovirus-mediated expression of human coagulation factor IX in the Rhesus macaque is associated with dose-limiting toxicity
47. Intravenous administration of recombinant adenoviruses causes thrombocytopenia, anemia and erythroblastosis in rabbits
48. Acute host-mediated endothelial injury after adenoviral gene transfer in normal rabbit arteries: Impact on transgene expression and endothelial function
49. Hematologic consequences of viral infections