Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

Molecular Therapy

Volumn 4, Issue 3, 2001, Pages 192-200

  Herzog, Roland W ^a   Mount, Jane D ^b   Arruda, Valder R ^a   High, Katherine A ^a   Lothrop Jr , Clinton D ^b  

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

^b AUBURN UNIVERSITY   (United States)

Author keywords

AAV; Factors IX; Gene transfer; Hemophilia B dogs; Muscle

Indexed keywords

BLOOD CLOTTING FACTOR 9; CYCLOPHOSPHAMIDE; MESNA; MESSENGER RNA;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLEEDING DISORDER; BLOOD CLOTTING; CONTROLLED STUDY; DOG; DOSE RESPONSE; DRUG SAFETY; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HEMOPHILIA B; IMMUNOMODULATION; IMMUNOSUPPRESSIVE TREATMENT; MALE; MISSENSE MUTATION; NONHUMAN; PARAMETER; PROTEIN EXPRESSION; PUBLICATION; SKELETAL MUSCLE; STOP CODON; X CHROMOSOME LINKAGE;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS;

EID: 0034849318     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0442     Document Type: Article

References (27)

1. Gene therapy for disorders of hemostasis
2. Gene therapy for the hemophilias
3. Complete short-term correction of canine hemophilia A by in vivo gene therapy
4. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
5. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
6. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
7. Factor IX: Molecular structure, epitopes, and mutations associated with inhibitor formation
8. Persistent expression of canine factor IX in hemophilia B canines
9. 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
10. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
11. A deletion mutation causes hemophilia B in Lhasa Apso dogs
12. Posttranslational modifications of recombinant myotube-synthesized human factor IX
13. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
14. Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy
15. Canine hemophilia B resulting from a point mutation with unusual consequences
16. Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony
17. Immune responses in hemophilia B mice expressing a species-specific transgene
18. A coagulation factor IX-deficient mouse model for human hemophilia B
19. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
20. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9
21. Factor IX inhibitors and anaphylaxis in hemophilia B
22. Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice
23. Induction of split tolerance and clinical cure in high-responding hemophiliacs with factor IX antibodies
24. Cyclophosphamide-induced immunological tolerance: An overview
25. Adeno-associated virus vectors can be efficiently produced without helper virus
26. The molecular basis of canine pyruvate kinase deficiency
27. Anti-factor VIII inhibitors and lupus anticoagulants in haemophilia A patients