Parvovirus-mediated gene transfer for the haemophilias

Haemophilia

Volumn 8, Issue SUPPL. 2, 2002, Pages 60-67

  Walsh, C E ^a   Chao, H ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus (AAV); Factor IX; Factor VIII; Gene therapy; Gene transfer; Haemophilia

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; GENE TRANSFER; HEMOPHILIA; HOST CELL; HUMAN; IMMUNE RESPONSE; MOUSE; NONHUMAN; PARVOVIRUS; PRIORITY JOURNAL; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS INFECTION;

BLOTTING, WESTERN; DEPENDOVIRUS; FACTOR IX; FACTOR VIII; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMOPHILIA A; HUMANS; SEROTYPING;

EID: 0036264622     PISSN: 13518216     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1351-8216.2001.00121.x     Document Type: Article

References (57)

1. Advances toward gene therapy for haemophilia at the millennium
2. Structure, biology, and genetics of factor VIII
3. The molecular basis of haemophilia B
4. Optimization of the human factor VIII complementary DNA expression plasmid for gene therapy of haemophilia A
5. Clinical aspects and therapy for haemophilia B
6. Stable gene transfer and expression of human coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
7. In vivo gene therapy of haemophilia B. sustained partial correction of factor IX-deficient dogs
8. Persistent expression of canine factor IX in haemophilia B canines
9. Long-term expression of human coagulation factor VIII and correction of haemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
10. Stage I clinical trial of gene therapy for haemophilia B
11. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
12. Non-viral gene transfer of blood caogulation factor VIII in patients with severe haemophilia A
13. Molecular biology of adeno-associated viruses
14. Recombinant human parvovirus B19 vectors: Erythroid cell-specific delivery and expression of transduced genes
15. Analysis of the cell and erythrocyte binding activities of the dimple and canyon regions of the canine parvovirus capsid
16. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles
17. Adeno-associated virus (AAV) type 5 Rep protein cleaves a unique terminal resolution site compared with other AAV serotypes
18. Adeno-associated virus (AAV) -3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors
19. Gene therapy vectors based on adeno-associated virus type 1
20. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2
21. Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells
22. Adeno-associated viral vectors
23. Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors
24. Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro
25. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
26. Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo
27. Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors
28. Immune responses to adenovirus and adeno-associated virus in humans
29. Building a better vector: The manipulation of AAV virions
30. Use of adeno-associated virus as a general transduction vector for mammalian cells
31. Genetics of adeno-associated virus: Isolation and preliminary characterization of adeno-associated virus type 2 mutants
32. Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression
33. New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors
34. High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type 1 vector expressing AAV-2 Rep and Cap
35. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain
36. Recombinant adeno-associated virus for muscle directed gene therapy
37. rAAV vector-mediated sarcoglycan gene transfer in a hamster model for limb girdle muscular dystrophy
38. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of haemophilia
39. Defective adeno-associated viral-mediated transfection of insulin gene by direct injection into liver parenchyma decreases blood glucose of diabetic mice
40. Sustained correction of bleeding disorder in haemophilia B mice by gene therapy
41. The kinetics of rAAV integration in the liver
42. Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle
43. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
44. Serologic evidence for human infection with adenovirus-associated viruses
45. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
46. Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant celltypes and regions in the mammalian central nervous system
47. Cloning and characterization of adeno-associated virus type 5
48. Adeno-associated virus type 2 binds to a 150-kilodalton cell membrane glycoprotein
49. The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers
50. Several log increase in therapeutic transgene delivery by district adeno-associated viral serotype vectors
51. Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV)
52. Repeated delivery of adeno-associated virus vectors to the rabbit airway
53. Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure
54. Detection of adeno-associated virus type 2 in human peripheral blood cells
55. Sustained and complete phenotypic correction of haemophilia B mice following intramuscular injection of AAVI sertotype vectors
56. Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
57. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer