Lentivirus vectors: Difficulties and hopes before clinical trials

Current Opinion in Molecular Therapeutics

Volumn 3, Issue 4, 2001, Pages 316-326

  Kafri, T ^a  

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

Author keywords

Central nervous system; Gene therapy; HIV; Lentivirus; Pseudotyping; Stem cells

Indexed keywords

ABC TRANSPORTER; ALANINE AMINOTRANSFERASE; BETA GLOBIN; BROMODEOXYCYTIDINE; CD34 ANTIGEN; COMPLEMENTARY DNA; CYTOKINE; DOXYCYCLINE; FIBRONECTIN; GAG PROTEIN; GREEN FLUORESCENT PROTEIN; IMMUNOGLOBULIN ENHANCER BINDING PROTEIN; NEF PROTEIN; ONCOPROTEIN; PHOSPHOGLYCERATE KINASE; POL PROTEIN; PROTEIN PRECURSOR; REV PROTEIN; SYNAPTOTAGMIN; TETRACYCLINE; TRANSACTIVATOR PROTEIN; UNINDEXED DRUG; VIF PROTEIN; VIRUS DNA; VIRUS ENVELOPE PROTEIN; VIRUS GLYCOPROTEIN; VIRUS PROTEIN; VIRUS RNA; VIRUS VECTOR; VPR PROTEIN; VPU PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BETA THALASSEMIA; CELL DIVISION; CLINICAL RESEARCH; CONTROLLED STUDY; DNA VECTOR; DRUG EFFICACY; DRUG SAFETY; GENE EXPRESSION; GENE TARGETING; GENE TECHNOLOGY; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; GENOME; HOST CELL; HUMAN; HUMAN CELL; HUMAN EXPERIMENT; IMMUNE RESPONSE GENE; LENTIVIRINAE; MOUSE; NONHUMAN; PARKINSON DISEASE; PHENOTYPE; RETROVIRUS; REVIEW; SPECIES DIFFERENCE; TARGET CELL; TRANSGENE;

BRAIN; CLINICAL TRIALS; GENE THERAPY; GENE TRANSFER, HORIZONTAL; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS; LENTIVIRUS; TRANSGENES;

EID: 0034890776     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (101)

1. HIV-1: Fifteen proteins and an RNA
2. Human immunodeficiency virus infection of cells arrested in the cell cycle
3. In vivo gene delivery and stable transduction of non-dividing cells by a lentiviral vector
4. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector
5. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
6. Kinetic analyses of stability of simple and complex retroviral vectors
7. Critical factors influencing stable transduction of human CD34(+) cells with HIV-1-derived lentiviral vectors
8. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector
9. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
10. Gene transfer to human pancreatic endocrine cells using viral vectors
11. Lentivirus-mediated transduction of islet grafts with interleukin 4 results in sustained gene expression and protection from insulitis
12. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer
13. Bcl-xL protects adult septal cholinergic neurons from axotomized cell death
14. Development of a self-inactivating lentivirus vector
15. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
16. A third-generation lentivirus vector with a conditional packaging system
17. Minimal requirement for a lentivirus vector based on human immunodeficiency virus type 1
18. A packaging cell line for lentivirus vectors
19. A replication-competent retrovirus arising from a split-function packaging cell line was generated by recombination events between the vector, one of the packaging constructs, and endogenous retroviral sequences
20. Lentivirus vector mobilization and spread by human immunodeficiency virus
21. Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus
22. Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells
23. Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system
24. Efficient transduction of non-dividing cells by optimized feline immunodeficiency virus vectors
25. Feline immunodeficiency virus vectors persistently transduce non-dividing airway epithelia and correct the cystic fibrosis defect
26. Minimum requirements for efficient transduction of dividing and non-dividing cells by feline immunodeficiency virus vectors
27. Gene transfer vectors derived from equine infectious anemia virus
28. Gene delivery to murine trachea by an equine lentiviral vector
29. Stable gene transfer to the nervous system using a non-primate lentiviral vector
30. Construction and molecular analysis of gene transfer systems derived from bovine immunodeficiency virus
31. Simian immunodeficiency virus RNA is efficiently encapsidated by human immunodeficiency virus type 1 particles
32. Primate and feline lentivirus vector RNA packaging and propagation by heterologous lentivirus virions
33. Species-specific, postentry barriers to primate immunodeficiency virus infection
34. A Rev-independent human immunodeficiency virus type 1 (HIV-1)-based vector that exploits a codon-optimized HIV-1 gag-pol gene
35. Modified human immunodeficiency virus-based lentiviral vectors display decreased sensitivity to trans-dominant Rev
36. Anti-human immunodeficiency virus type 1 gene therapy in human central nervous system-based cells: An initial approach against a potential viral reservoir
37. Development of a novel trans-lentiviral vector that affords predictable safety
38. Integration complexes derived from HIV vectors for rapid assays in vitro
39. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors
40. A stable system for the high-titer production of multiply attenuated lentiviral vectors
41. An inducible packaging cell system for safe, efficient lentiviral vector production in the absence of HIV-1 accessory proteins
42. Lentikat3™: A novel third generation, stable packaging cell line for Lentivirus production
43. A second origin of DNA plus-strand synthesis is required for optimal human immunodeficiency virus replication
44. HIV-1 reverse transcription. A termination step at the center of the genome
45. HIV-1 genome nuclear import is mediated by a central DNA flap
46. The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain
47. The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells
48. Enhanced transgene expression in cord blood CD34(+)-derived hematopoietic cells, including developing T-cells and nod/scid mouse repopulating cells, following transduction with modified trip lentiviral vectors
49. Transduction of liver cells by lentiviral vectors: Analysis in living animals by fluorescence imaging
50. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences
51. Lentivirus-mediated gene transfer in primary T-cells is enhanced by a central DNA flap
52. Lentiviral vectors: Regulated gene expression
53. Development of multigene and regulated lentivirus vectors
54. Development of second generation inducible lentiviral vectors
55. In vivo regulation of transgene expression in tumor and hematopoiesis by new lentiviral vectors
56. Immunogenicity of tetracycline-dependent transcription factor in monkeys and its impact on reporter gene expression
57. Pseudotyping human immunodeficiency virus type 1 (HIV-1) by the glycoprotein of vesicular stomatitis virus targets HIV-1 entry to an endocytic pathway and suppresses both the requirement for Nef and the sensitivity to cyclosporin A
58. Human immunodeficiency virus type 1 particles pseudotyped with envelope proteins that fuse at low pH no longer require Nef for optimal infectivity
59. Separable mechanisms of attachment and cell uptake during retrovirus infection
60. VSV-G pseudotyped lentiviral vector particles produced in human cells are inactivated by human serum
61. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver
62. Impact of preexisting and induced humoral and cellular immune responses in an adenovirus-based gene therapy phase I clinical trial for localized mesothelioma
63. Sequences in the cytoplasmic tail of the gibbon ape leukemia virus envelope protein that prevent its incorporation into lentivirus vectors
64. Lentiviral vectors pseudotyped with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1
65. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo
66. 1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T-cells
67. Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent
68. Efficient lentiviral transduction of liver requires cell cycling in vivo
69. Multiply attenuated lentiviral vector acheives efficient gene delivery in vivo
70. Lentiviral-mediated gene transfer into human lymphocytes: Role of HIV-1 accessory proteins
71. Gene transfer into stimulated and unstimulated T-lymphocytes by HIV-1-derived lentiviral vectors
72. HIV-flap mediated gene therapy for adrenoleukodystrophy: Efficient correction of hematopoietic stem cells using short clinically applicable protocole
73. Gene therapy for hemophilia A using lentiviral vectors
74. Expansion of human cord blood CD34(+)CD38(-) cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: Dissociation of SRC phenotype and function
75. Fibronectin fragment CH-296 inhibits apoptosis and enhances ex vivo gene transfer by murine retrovirus and human lentivirus vectors independent of viral tropism in non-human primate CD34(+) cells
76. Transduction of human CD34+ CD38-bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors
77. Marking and gene expression by a lentivirus vector in transplanted human and non-human primate CD34(+) cells
78. Efficient human immunodeficiency virus-based vector transduction of unstimulated human mobilized peripheral blood CD34+ cells in the SCID-hu Thy/Liv model of human T-cell lymphopoiesis
79. Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice
80. Stable transduction with lentiviral vectors and amplification of immature hematopoietic progenitors from cord blood of preterm human fetuses
81. Lentivirus gene transfer in murine hematopoietic progenitor cells is compromised by a delay in proviral integration and results in transduction mosaicism and heterogeneous gene expression in progeny cells
82. Prolonged high-level detection of retrovirally marked hematopoietic cells in non-human primates after transduction of CD34+ progenitors using clinically feasible methods
83. High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors
84. A murine leukemia virus (MuLV) long terminal repeat derived from rhesus macaques in the context of a lentivirus vector and MuLV gag sequence results in high-level gene expression in human T-lymphocytes
85. Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression
86. Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector
87. Lentivirus-mediated gene transfer of gp91phox corrects chronic granulomatous disease (CGD) phenotype in human X-CGD cells
88. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
89. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector
90. Lentiviral vectors as a gene delivery system in the mouse midbrain: Cellular and behavioral improvements in a 6-OHDA model of Parkinson's disease using GDNF
91. Lentiviral gene transfer to the non-human primate brain
92. Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease
93. Adeno-associated virus and lentivirus vectors mediate efficient and sustained transduction of cultured mouse and human dorsal root ganglia sensory neurons
94. Constitutive NF-κB activity in neurons
95. Intercellular trafficking and protein delivery by a herpesvirus structural protein
96. Transduction of full-length TAT fusion proteins into mammalian cells: TAT-p27Kip1 induces cell migration
97. Intercellular delivery of a herpes simplex virus VP22 fusion protein from cells infected with lentiviral vectors
98. Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer
99. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: Correction of neuropathology and protection against learning impairments in affected mice
100. Towards a neuroprotective gene therapy for Parkinson's disease: Use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in the rat Parkinson model
101. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease