Challenges and strategies for cystic fibrosis lung gene therapy

Molecular Therapy

Volumn 4, Issue 2, 2001, Pages 84-91

  Koehler, David R ^a,b   Hitt, Mary M ^c   Hu, Jim ^a,b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c MCMASTER UNIVERSITY   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; LIPOSOME; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS VECTOR;

ADENOVIRUS; AIRWAY; CYSTIC FIBROSIS; DRUG DELIVERY SYSTEM; DRUG FORMULATION; EXPRESSION VECTOR; GENE CASSETTE; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HUMAN; LUNG FIBROSIS; MEDICAL RESEARCH; NONHUMAN; REVIEW; TRANSGENE;

EID: 0034891448     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0435     Document Type: Review

References (54)

1. Gene therapy for lung disease: Hype or hope?
2. Cystic Fibrosis
3. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction
4. Role of CFTR in airway disease
5. Marked enhancement of direct respiratory tissue transfection by aurintricarboxylic acid
6. Targeting transgene expression for cystic fibrosis gene therapy
7. Factors controlling the efficiency of cationic lipid-mediated transfection in vivo via intravenous administration
8. Biodistribution and transgene expression with nonviral cationic vector/DNA complexes in the lungs
9. In vivo protein transduction: Delivery of a biologically active protein into the mouse
10. Apical localization of the coxsackievirus and adenovirus receptor by GPI modification is sufficient for adenovirus-mediated gene transfer from the apical surface of human airway epithelia
11. Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for inefficient gene transfer
12. Targeting adenovirus
13. Modification of an adenoviral vector with biologically selected peptides: A novel strategy for gene delivery to cells of choice
14. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer
15. Increasing epithelial junction permeability enhances gene transfer to airway epithelia in vivo
16. Instillation of perfluorochemical (PFC) liquid to lungs of spontaneously breathing mice transiently opens tight junctions between airway epithelial cells: A possible mechanism for enhancement of gene expression using PFC liquids
17. Mechanism by which calcium phosphate coprecipitation enhances adenovirus-mediated gene transfer
18. Potentiation of gene transfer to the mouse lung by complexes of adenovirus vector and polycations improves therapeutic potential
19. Characterization of cationic liposome-mediated gene transfer in vivo by intravenous administration
20. Gene delivery mediated by cationic liposomes: From biophysical aspects to enhancement of transfection
21. Metabolic instability of plasmid DNA in the cytosol: A potential barrier to gene transfer
22. Size-dependent DNA mobility in cytoplasm and nucleus
23. Cellular and molecular barriers to gene transfer by a cationic lipid
24. A nuclear localization signal can enhance both the nuclear transport and expression of 1 kb DNA
25. Nuclear targeting peptide scaffolds for lipofection of nondividing mammalian cells
26. Pulmonary inflammation induced by incomplete or inactivated adenoviral particles
27. Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo
28. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors
29. Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung
30. Long-term hepatic adenovirus-mediated gene expression in mice following CTLA41g administration
31. Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs
32. "Stealth" adenoviruses blunt cell-mediated and humoral immune responses against the virus and allow for significant gene expression upon readministration in the lung
33. Polymer-coated adenovirus permits efficient retargeting and evades neutralising antibodies
34. "Sero-switch" adenovirus-mediated in vivo gene transfer: Circumvention of anti-adenovirus humoral immune defenses against repeat adenovirus vector administration by changing the adenovirus serotype
35. Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype
36. Extensive cross-reactivity of adenovirus-specific cytotoxic T cells
37. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy
38. Elimination of both E1 and E2 from adenovirus vectors further improves prospects for in vivo human gene therapy
39. Long-term gene delivery into the livers of immunocompetent mice with E1/E4-defective adenoviruses
40. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene
41. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
42. Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes
43. Efficient CFTR expression from AAV vectors packaged with promoters-the second generation
44. Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:pDNA complexes
45. A Toll-like receptor recognizes bacterial DNA
46. A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis
47. Functional consequences of heterologous expression of the cystic fibrosis transmembrane conductance regulator in fibroblasts
48. CFTR modulates lung secretory cell proliferation and differentiation
49. Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR
50. Human cystic fibrosis transmembrane conductance Regulator directed to respiratory epithelial cells of transgenic mice
51. Characterization of the cystic fibrosis transmembrane conductance regulator promoter region. Chromatin context and tissue-specificity
52. Targeting transgene expression to airway epithelia and submucosal glands, prominent sites of human CFTR expression
53. Promoter attenuation in gene therapy: Interferon-γ and tumor necrosis factor-α inhibit transgene expression
54. TNF-α and IFN-γ induced by innate anti-adenoviral immune responses inhibit adenovirus-mediated transgene expression