Factors affecting long-term expression of a secreted transgene product after intravenous administration of a retroviral vector

Molecular Therapy

Volumn 3, Issue 4, 2001, Pages 516-525

  McCormack, James E ^a,b   Edwards, Wilson ^a,c   Sensintaffer, John ^a,d   Lillegren, Larisa ^a,e   Kozloski, Melinda ^a,f   Brumm, Duane ^a,f   Karavodin, Linda ^a,e   Jolly, Douglas J ^a,g   Greengard, Judith ^a,c  

^a NOVARTIS PHARMA AG   (Switzerland)

^b Paik Inje Memorial Institute   (United States)

^c AMGEN INC   (United States)

^d PFIZER INC   (United States)

^e Maxim Pharmaceuticals Inc   (United States)

^f Gen Probe Incorporated   (United States)

^g AGILENT TECHNOLOGIES   (United States)

Author keywords

Growth hormone; Intravenous; Long term expression; Retroviral vector

Indexed keywords

BLOOD CLOTTING FACTOR 8; GENE PRODUCT; HUMAN GROWTH HORMONE; VIRUS VECTOR;

AGE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL DIVISION; CIRCULATION; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; GROWTH HORMONE BLOOD LEVEL; IMMUNE RESPONSE; IMMUNOMODULATION; IN VIVO STUDY; LIVER; MODEL; MOUSE; NONHUMAN; PARAMETER; RECIPIENT; RETROVIRUS; SPLEEN; TRANSGENE;

EID: 0034987586     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0259     Document Type: Article

References (53)

1. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits
2. Retroviral-mediated gene transfer into hepatocytes in vivo
3. In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs
4. T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
5. Gene therapy via primary myoblasts: Long-term expression of factor IX protein following transplantation in vivo
6. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
7. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
8. Retroviral vectors
9. Human gene marker/therapy clinical protocols
10. Animal testing of retroviral-mediated gene therapy for factor VIII deficiency
11. Systematic expression of human factor VIII from peripheral retroviral gene delivery in rabbits and dogs
12. Intravenous retroviral vectors expressing human factor VIII results in long-term expression in rabbits of various age groups
13. Phase I trial of FVIII gene transfer using a retroviral vector administered by peripheral intravenous infusion: Interim safety update
14. Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase
15. Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes
16. Portal branch occlusion safely facilitates in vivo retroviral vector transduction of rat liver
17. Tri-iodothyronine and a deleted form of hepatocyte growth factor act synergistically to enhance liver proliferation and enable in vivo retroviral gene transfer via the peripheral venous system
18. Advances toward gene therapy for hemophilia at the Millennium
19. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production
20. Human growth hormone: Complementary DNA cloning and expression in bacteria
21. Cross-reactive lysis of human targets infected with prototypic and clinical human immunodeficiency virus type 1 (HIV-1) strains by murine anti-HIV-1 IIIB env-specific cytotoxic T lymphocytes
22. Direct injection of a recombinant retroviral vector induces human immunodeficiency virus-specific immune responses in mice and nonhuman primates
23. Establishment of parameters for optimal transduction efficiency and antitumor effects with purified high-titer HSV-TK retroviral vector in established solid tumors
24. Generation of a systemic antitumor response with regional intratumoral injections of interferon gamma retroviral vector
25. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production
26. Evidence for localization of biologically active recombinant retroviral vector to lymph nodes in mice injected intramuscularly
27. Preparation of extracts from yeast
28. Anti-vector immunoglobulin induced by retroviral vectors
29. Metallothionein-human GH fusion genes stimulate growth of mice
30. Insulin antibodies and the control of diabetes
31. Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy
32. Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice
33. Plasma factor VIII synthesis and control as assessed by canine organ transplantation
34. Correction of coagulation in the hemophilic dog by transplantation of lymphatic tissue
35. Method for multiple portal vein infusions in mice: Quantitation of adenovirus-mediated hepatic gene transfer
36. Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs
37. Transient immunosuppression allows transgene expression following readministration of adeno-associated viral vectors
38. Evaluation of PCR and ELISA assays for screening clinical trial subjects for replication-competent retroviruses
39. Delayed-type hypersensitivity response to high doses of adenoviral vectors
40. Ovarian cancer BRCA1 gene therapy: Phase I and II trial differences in immune response and vector stability
41. Effective repeat administration with adenovirus vectors to the muscle
42. Expression and characterization of human CD4:immunoglobulin fusion proteins
43. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII
44. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
45. Complete short-term correction of canine hemophilia A by in vivo gene therapy
46. Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung
47. The immunosuppressant FK506 prolongs transgene expression in brain following adenovirus-mediated gene transfer
48. Immunomodulation with FK506 around the time of intravenous re-administration of an adenoviral vector facilitates gene transfer into primed rat liver
49. Prevention of immune reactions triggered by first-generation adenoviral vectors by monoclonal antibodies and CTLA41g
50. Readministration of adenovirus vector in nonhuman primate lungs by blockade of CD40-CD40 ligand interactions
51. Transient immunomodulation with anti-CD40 ligand antibody and CTLA41g enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver
52. Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinemia type 1