Gene Therapy Vectors As Drug Delivery Systems

Clinical Pharmacokinetics

Volumn 28, Issue 3, 1995, Pages 181-189

  Afione, Sandra A ^a   Conrad, Carol K ^a   Flotte, Terence R ^a,b  

^a JOHNS HOPKINS UNIVERSITY   (United States)

^b JOHNS HOPKINS HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LIPOSOME;

ADENOVIRUS; CELL NUCLEUS; CLONING VECTOR; DRUG DELIVERY SYSTEM; EDITORIAL; GENE TARGETING; GENE THERAPY; NONHUMAN; PARVOVIRUS; PRIORITY JOURNAL; RETROVIRUS;

DRUG DELIVERY SYSTEMS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMAN; SUPPORT, NON-U.S. GOV'T; SUPPORT, U.S. GOV'T, P.H.S.;

EID: 0028912789     PISSN: 03125963     EISSN: 11791926     Source Type: Journal    
DOI: 10.2165/00003088-199528030-00001     Document Type: Article

References (104)

1. Progress toward human gene therapy
2. Cystic fibrosis
3. 5 promote adenovirus internalization but not virus attachment
4. High-efficiency gene transfer mediated by adenovirus-polylysine-DNA complexes
5. Adenoviridae and their replication
6. Binding-incompetent adenovirus facilitates molecular conjugate-mediated gene transfer by the receptor-mediated endocytosis pathway
7. Retroviridae and their replication
8. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection
9. Retrovirusmediated transfer of the human γ-IFN gene: a therapy for cancer
10. Altering the genome by homologous recombination
11. Regulation of somatic-cell therapy and gene therapy by the Food and Drug Administration
12. Gene transfer into humans — immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction
13. Correction of ADA deficiency in human T-lympocytes using retroviral mediated gene transfer
14. Development of gene therapy for immunodeficiency: adenosine deaminase deficiency
15. Lymphocytes as cellular vehicles for gene therapy in mouse and man
16. Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene
17. Retrovirusmediated transduction of adult hepatocytes
18. Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit
19. Ex vivo gene therapy of familial hypercholesterolemia
20. Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolemia
21. In vivo gene therapy of hemophilia B: sustained partial correction in factor DC-deficient dogs
22. Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilie dogs
23. An alternative approach to somatic cell gene therapy
24. Development of retrovirus vectors useful for expressing genes in cultured murine embryonal cells and hematopoietic cells in vivo
25. Development of a high-titer retrovirus producer cell line and strategies for retrovirus-mediated gene transfer into Rhesus monkey hematopoietic stem cells
26. Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo
27. Retroviralmediated transfer and expression of human beta-globin genes in cultured murine and human erythroid cells
28. Retroviral vectors for the beta-globin gene that demonstrate improved titer and expression
29. Retroviral vectors for use in human gene therapy for cancer, Gaucher disease, and arthritis
30. Adenovirus-mediated transfer of a recombinant alpha1-antitrypsin gene to the lung epithelium in vivo
31. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium
32. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
33. Adenovirus-mediated transfer of the CFTR gene to lung of non-human primates: biological efficacy study
34. Gene therapy for cystic fibrosis using E1-deleted adenovirus: a phase I trial in the nasal cavity
35. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis
36. Direct gene transfer for the understanding and treatment of human disease
37. Direct gene transfer with DNA liposome complexes in humans: expression, lack of toxicity and therapeutic potential in melanoma
38. Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus
39. High-efficiency gene transfer into mammalian cells: generation of helper-free recombinant retrovirus with broad mammalian host range
40. Introduction of a selectable gene into different animal tissue by a retrovirus recombinant vector
41. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges
42. Retroviral gene transfer using safe and efficient packaging cell lines
43. Insertion of the bacterial ‘gpt’ gene into the germ line of mice by retroviral insertion
44. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer
45. Gene transfer of drug resistance genes: implications for cancer therapy
46. Rescue of type I collagendeficient phenotype by retroviral-vector-mediated transfer of human pro alpha 1 (I) collagen gene into mov-13 cells
47. Gene-marking to trace origin of relapse after autologous bone-marrow transplantation
48. In situ retroviral mediated gene transfer for the treatment of brain tumors in rats
49. In vivo gene transfer with retroviral vector-producer cells for treatment of experimental brain tumors
50. Characteristics of a human cell line transformed by DNA from adenovirus type 5
51. Adenovirus-mediated in vivo gene transfer and expression in normal rat liver
52. Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: toxicity study
53. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy
54. Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver
55. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis
56. An approach to treating the hepatobiliary disease of cystic fibrosis by somatic gene transfer
57. Adenovirus-mediated transfer of a recombinant human α-antitrypsin cDNA to human endothelial cells
58. Gene therapy for vascular smooth muscle cell proliferation after arterial injury
59. An adenovirus vector for gene transfer into neurons and glia in the brain
60. In vivo adenovirus-mediated gene transfer into ocular tissues
61. Adeno-associated virus vectors
62. Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter
63. Site-specific integration by adeno-associated virus
64. Targeted integration of adeno-associated virus (AAV) into human chromosome 19
65. Characterization of a preferred site on chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination
66. Adeno-associated virus: integration at a specific chromosomal locus
67. Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA
68. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression
69. Gene expression from adeno-associated virus vectors in airway epithelial cells
70. An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction
71. Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors
72. Adeno-associated virus vector gene expression occurs in non-dividing cells in the absence of vector DNA integration
73. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells
74. Adeno-associated virus vector for high-frequency integration, expression and rescue of genes in mammalian cells
75. A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase
76. Recombinant adenoassociated virus-mediated gene transfer into hematopoietic progenitor cells
77. Regulated high level expression of a human γ-globin gene introduced into erythroid cells by an adeno-associated virus vector
78. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter
79. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector
80. − channels by protein kinase A corrected by insertion of CFTR
81. Both CFTR and outwardly rectifying chloride channels contribute to cAMP-stimulated whole cell chloride currents
82. Dual-target inhibition of HIV-1 by means of an adeno-associated virus antisense vector
83. Suppression of human α-globin gene expression mediated by recombinant adeno-associated virus 2-based antisense vectors
84. Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells
85. Administration of AAV-CFTR vectors to the airway epithelium of non-human primates is safe and efficacious
86. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure
87. Gene transfer by lipofection in rabbit and human secretory epithelial cells
88. Cationic liposomes for direct gene transfer in therapy of cancer and other diseases
89. Direct injection of hepatitis B virus DNA into liver induced hepatitis in adult rats
90. Efficient and sustained gene expression in primary T lymphocytes and primary cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cationic liposomes
91. Localization and induced expression of fusion genes in the rat lung
92. Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer
93. Systemic gene expression after intravenous DNA delivery into adult mice
94. Expression of human growth hormone fusion genes in cultured lung endothelial cells and in the lungs of mice
95. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy
96. Protection of rabbit lungs from endotoxin injury by in vivo hyper-expression of the prostaglandin G/H synthase gene
97. Aerosol and intravenous transfection of human α1-antitrypsin gene to lungs of rabbits
98. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice
99. Gene therapy for cystic fibrosis in humans by liposome-mediated DNA transfer: the production of resources and the regulatory process
100. Gene transfer to respiratory epithelial cells via the receptor-mediated endocytosis pathway
101. Receptor-mediated gene transfer to airway epithelial cells in primary culture
102. Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptor-mediated gene transfer
103. Regulation of the phoshoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer
104. Gene transfer in respiratory epithelial cells by targeting the polymeric immunoglobulin receptor