Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9

Molecular Therapy

Volumn 4, Issue 3, 2001, Pages 201-210

  Fields, Paul A ^a   Arruda, Valder R ^a   Armstrong, Elina ^a   Chu, Kirk ^a   Mingozzi, Federico ^a   Hagstrom, J Nathan ^a   Herzog, Roland W ^a   High, Katherine A ^a  

^a HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

AAV; Antibody; Factor IX; Hemophilia B; Immune modulation; Muscle

Indexed keywords

BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 CONCENTRATE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; CYTOTOXIC T LYMPHOCYTE ANTIGEN 4; GENE PRODUCT; GLYCOPROTEIN GP 39; IMMUNOGLOBULIN; MONOCLONAL ANTIBODY; PROTEIN ANTIBODY; TACROLIMUS; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY PRODUCTION; ARTICLE; BLEEDING DISORDER; CONTROLLED STUDY; DISEASE SEVERITY; DRUG SAFETY; GENE DELETION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HEMOPHILIA B; HUMORAL IMMUNITY; IMMUNE RESPONSE; IMMUNOMODULATION; IMMUNOSUPPRESSIVE TREATMENT; MOUSE; NONHUMAN; TH2 CELL; TRANSGENE; X CHROMOSOME LINKAGE;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 0034853102     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0441     Document Type: Article

References (34)

1. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
2. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
3. Recombinant adeno-associated virus for muscle directed gene therapy
4. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
5. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers
6. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
7. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
8. Factor IX: Molecular structure, epitopes, and mutations associated with inhibitor formation
9. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
10. The hemophilias: Progress and problems
11. Factor IX inhibitors and anaphylaxis in hemophilia B
12. A coagulation factor IX-deficient mouse model for human hemophilia B
13. The molecular basis of hemophilia A and B
14. Recombinant human factor IX: Replacement therapy, prophylaxis, and pharmacokinetics in canine hemophilia B
15. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
16. Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung
17. Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA4Ig
18. Transient subversion of CD40 ligand function diminishes immune responses to adenovirus vectors in mouse liver and lung tissues
19. CD40 ligand-dependent T cell activation: Requirement of B7-CD28 signaling through CD40
20. Immunosuppression through blockade of CD28:B7-mediated costimulatory signals
21. Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver
22. Prevention and treatment of factor VIII inhibitors in murine hemophilia A
23. Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration
24. CD28-B7 T cell costimulatory blockade by CTLA4Ig in the rat renal allograft model: Inhibition of cell-mediated and humoral immune responses in vivo
25. Adenovirus-mediated gene transfer into cold-preserved liver allografts: Survival pattern and unresponsiveness following transduction with CTLA4Ig
26. Cyclophosphamide-induced immunological tolerance: An overview
27. Natural adjuvants: Endogenous activators of dendritic cells
28. A deletion mutation causes hemophilia B in Lhasa Apso dogs
29. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B cause by a null mutation
30. Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter
31. Adeno-associated virus vectors can be efficiently produced without helper virus
32. Biochemical characterization of recombinant factor IX
33. Human factor IX corrects the bleeding diathesis of mice with hemophilia B
34. 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