Gene transfer into nonhuman primate hematopoietic stem cells: Implications for gene therapy

Stem Cells

Volumn 19, Issue 1, 2001, Pages 12-23

  Hanazono, Yutaka ^a   Terao, Keiji ^b   Ozawa, Keiya ^a  

^a JICHI MEDICAL UNIVERSITY   (Japan)

^b NATIONAL INSTITUTE OF INFECTIOUS DISEASES   (Japan)

Author keywords

Gene therapy; Hematopoietic stem cells; Nonhuman primate models; Retroviral vectors

Indexed keywords

BETA GLOBIN; FIBRONECTIN; FLT3 LIGAND;

ANIMAL CELL; ANIMAL EXPERIMENT; CELL DIFFERENTIATION; CELL RENEWAL; DNA VECTOR; FELINE LEUKEMIA VIRUS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; GIBBON LEUKEMIA VIRUS; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; MARKER GENE; NONHUMAN; PRIMATE; RECEPTOR DENSITY; RETROVIRUS; REVIEW; STEM CELL TRANSPLANTATION; VIRUS ENVELOPE;

ALOCASIA MACRORRHIZOS; ANIMALIA; FELIDAE; FELINE LEUKEMIA VIRUS; GIBBON APE LEUKEMIA VIRUS; HYLOBATES SP.; MURINAE; PRIMATES; UNIDENTIFIED RETROVIRUS;

EID: 0035181593     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1634/stemcells.19-1-12     Document Type: Review

References (145)

1. Gene therapy using hematopoietic stem cells: Sisyphus approaches the crest
2. Gene therapy - Promises, problems and prospects
3. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
4. Adenovirus vector for gene therapy
5. Adeno-associated virus vectors and hematology
6. Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation
7. Genetic marking
8. Clinical trials of gene therapy using hematopoietic stem cells
9. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
10. Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells
11. Naturally occurring primary deficiencies of the immune system
12. Stable and functional lymphoid reconstitution of common cytokine receptor γ chain deficient mice by retroviral-mediated gene transfer
13. Evidence that hematopoiesis may be a stochastic process in vivo
14. Cell-surface receptors for retroviruses and implications for gene transfer
15. The level of mRNA encoding the amphotropic retrovirus receptor in mice and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction
16. Efficient transduction by an amphotropic retrovirus vector is dependent on the high-level expression of the cell surface virus receptor
17. Effect of changes in expression of the amphotropic retroviral receptor PiT-2 on transduction efficiency and viral titer: Implications for gene therapy
18. Retrovirus-mediated gene transduction into long-term repopulating marrow cells of dogs
19. Retrovirus-mediated gene transduction into canine peripheral blood repopulating cells
20. Long-term persistence of canine hematopoietic cells genetically marked by retrovirus vectors
21. Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine α-L-iduronidase deficiency
22. The common marmoset as a target preclinical primate model for cytokine and gene therapy studies
23. Transduction of feline hematopoietic cells by oncoretroviral vectors pseudotyped with the subgroup A feline leukemia virus (FeLV-A)
24. Primates
25. Gene transfer into hematopoietic stem cells of nonhuman primates
26. Advantages and limitations of nonhuman primates as animal models in genetic research on complex diseases
27. The importance of non-human primates for preclinical testing of immunosuppressive monoclonal antibodies
28. Use of human leukocyte-specific monoclonal antibodies for clinically immunophenotyping lymphocytes of rhesus monkeys
29. Flow cytometric analysis on reactivity of human T lymphocyte-specific and cytokine-receptor-specific antibodies with peripheral blood mononuclear cells of chimpanzee (Pan troglodytes), rhesus macaque (Macaca mulatta), and squirrel monkey
30. Nonhuman primate models for human disease
31. The Philippine cynomolgus monkey (Macaca fascicularis) provides a new nonhuman primate model of tuberculosis that resembles human disease
32. Comparison of research cost: Man - Primate animal - Other animal models
33. Assay systems for hematopoietic stem and progenitor cells
34. Persistence of human multilineage, self-renewing lymphohematopoietic stem cells in chimeric sheep
35. Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: Implications for gene therapy
36. A newly discovered class of human hematopoietic cells with SCID-repopulating activity
37. Amphotropic murine leukemia retrovirus is not an acute pathogen for primates
38. No retroviremia or pathology in long-term follow-up of monkeys exposed to a murine amphotropic retrovirus
39. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer
40. Characterization of a replication-competent retrovirus resulting from recombination of packaging and vector sequences
41. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
42. Minimal requirement for a lentivirus vector based on human immunodeficiency virus type 1
43. A third-generation lentivirus vector with a conditional packaging system
44. Development of a self-inactivating lentivirus vector
45. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
46. Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability
47. Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor
48. Consistent and high rates of gene transfer can be obtained using flow-through transduction over a wide range of retroviral titers
49. Improved methods of retroviral vector transduction and production for gene therapy
50. Centrifugal enhancement of retroviral mediated gene transfer
51. Increased gene transfer into human cord blood cells by centrifugation-enhanced transduction in fibronectin fragment-coated tubes
52. Retroviral-mediated gene transfer in human bone marrow cells growth in continuous perfusion culture vessels
53. Bone marrow extracellular matrix molecules improve gene transfer into human hematopoieric cells via retroviral vectors
54. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells
55. Improved gene transfer into baboon marrow repopulating cells using recombinant fibronectin fragment CH-296 in combination with intefleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor
56. Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells
57. Gene transfer into marrow repopulating cells: Comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons
58. Improved gene transfer into canine hematopoietic repopulating cells using CD34-enriched marrow cells in combination with a gibbon ape leukemia virus-pseudotype retroviral vector
59. A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus-pseudotyped retroviral vector
60. Cell-surface receptors for gibbon ape leukemia virus and amphotropic murine retrovirus are inducible sodium-dependent phosphate symporters
61. Inhibition of VSV binding and infectivity by phosphatidylserine: Is phosphatidylserine a VSV-binding site?
62. Characterization of membrane components of the erythrocyte involved in vesicular stomatitis virus attachment and fusion at acidic pH
63. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: Concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells
64. Pseudotyped retroviral vectors for studies of human gene therapy
65. One-day ex vivo culture allows effective gene transfer into human nonobese diabetic/severe combined immune-deficient repopulating cells using high-titer vesicular stomatitis virus G protein pseudotyped retrovirus
66. + hematopoietic cells using RD-114-pseudotyped oncoretroviruses
67. The RD114/simian type D retrovirus receptor is a neutral amino acid transporter
68. Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein
69. Development of lentiviral vectors for gene therapy for human diseases
70. + cells by a recombinant human immunodeficiency virus retroviral vector
71. Gene transfer into human lymphocytes by a defective human immunodeficiency virus type 1 vector
72. Efficient transduction of nondividing human cells by feline immunodeficiency lentiviral vectors
73. Gene transfer vectors derived from equine infectious anemia virus
74. Lentivirus vectors using human and simian immunodeficiency virus elements
75. Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus
76. Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system
77. Lentiviral vectors pseudotyped with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1
78. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
79. - bone marrow and cord blood-derived SCIDrepopulating cells with third-generation lentiviral vectors
80. + cells
81. Gene silencing as a threat to the success of gene therapy
82. Inactivation of a GFP retrovirus occurs at multiple levels in long-term repopulating stem cells and their progeny
83. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo
84. Spontaneous silencing of humanized green fluorescent protein (hGFP) gene expression from a retroviral vector by DNA methylation
85. Epigenetics: Regulation through repression
86. Sustained gene expression in retrovirally transduced, engrafting human hematopoietic stem cells and their lympho-myeloid progeny
87. Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector
88. High-efficiency transduction and long-term gene expression with a murine stem cell retroviral vector encoding the green fluorescent protein in human marrow stromal cells
89. - cells expanded in vitro: Cis elements of FMEV are superior to those of Mo-MuLV
90. Position-independent, high-level expression of the human β-globin gene in transgenic mice
91. Molecular analysis of the human β-globin locus activation region
92. Full activity from human β-globin locus control region trangenes requires 5′HS1, distal β-globin promoter, and 3' β-globin sequences
93. High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells
94. A human β-globin gene fused to the human β-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells
95. Development of viral vectors for gene therapy of β-chain hemoglobinopathies: Optimization of a γ-globin gene expression cassette
96. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
97. An insulator element and condensed chromatin region separate the chicken β-globin locus from an independently regulated erythroid-specific folate receptor gene
98. The protein CTCF is required for the enhancer blocking activity of vertebrate insulators
99. A chromatin insulator protects retrovirus vectors from chromosomal position effects
100. Cell-surface markers for assessing gene transfer into human hematopoietic cells
101. Sustained high-level reconstitution of the hematopoietic system by preselected hematopoietic cells expressing a transduced cell-surface antigen
102. Use of the green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells
103. Ex vivo expansion of hematopoietic stem and progenitor cells - Robbing Peter to pay Paul?
104. Ex vivo expansion of hematopoietic precursors, progenitors, and stem cells: The next generation of cellular therapeutics
105. Ex vivo expansion of hematopoietic stem and progenitor cells: Are we there yet?
106. Long-term ex vivo maintenance and expansion of transplantable human hematopoietic stem cells
107. - cells in ex vivo culture during retroviral transduction without a corresponding increase in SCD repopulating cell (SRC) frequency: Dissociation of SRC phenotype and function
108. CD34 positive PBPC expanded ex vivo may not provide durable engraftment following myeloablative chemoradiotherapy regimens
109. In vivo drug-selectable genes: A new concept in gene therapy
110. Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1
111. Results of MDR-1 vector modification trial indicate that granulocyte/macrophage colony-forming unit cells do not contribute to posttransplant hematopoietic recovery following intensive systemic therapy
112. Phase I trial of retroviral-mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem-cell transplantation
113. Engraftment of MDR1 and NeoR gene-transduced hematopoietic cells after breast cancer chemotherapy
114. Expression and activity of P-glycoprotein, a multidrug efflux pump, in human hematopoietic stem cells
115. Subpopulations of normal peripheral blood and bone marrow cells express a functional multidrug resistance phenotype
116. Cytokine prestimulation as a gene therapy strategy: Implications for using the MDR1 gene as a dominant selectable marker
117. Expression of retroviral vectors containing the human multidrug resistance 1 cDNA in hematopoietic cells of transplanted mice
118. Transduction of murine bone marrow cells with an MDR1 vector enables ex vivo stem cell expansion, but these expanded grafts cause a myeloproliferative syndrome in transplanted mice
119. Retroviral transduction and expression of the human alkyltransferase cDNA provides nitrosourea resistance to hematopoietic cells
120. 6-alkylguanine-DNA-alkyltransferase
121. A gene transfer strategy for making bone marrow cells resistant to trimetrexate
122. In vivo selection of retrovirally transduced hematopoietic stem cells
123. A proliferation switch for genetically modified cells
124. Development of a novel selective amplifier gene for controllable expansion of transduced hematopoietic cells
125. Expansion of genetically modified primary human hematopoietic cells using chemical inducers of dimerization
126. In vivo selection using a cellgrowth switch
127. Retroviral marking and transplantation of rhesus hematopoietic cells by nonmyeloablative conditioning
128. Efficient and durable gene marking of hematopoietic progenitor cells in nonhuman primates after nonablative conditioning
129. Mild preconditioning and low-level engraftment confer methotrexate resistance in mice transplanted with marrow expressing drug-resistant dihydrofolate reductase activity
130. 6-methylguanine-DNA methyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection
131. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors
132. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients
133. Herpes simplex virus thymidine kinase gene transfer for controlled graft-versus-host disease and graft-versus-leukemia: Clinical follow-up and improved new vectors
134. Immune response to green fluorescent protein: Implications for gene therapy
135. Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model
136. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow cells
137. + bone marrow cells
138. + hematopoietic cell transduction with cell-free retroviral vector preparations
139. Long-term in vivo expression of the MFG-ADA retroviral vector in rhesus monkeys transplanted with transduced bone marrow cells
140. Developmental potential and dynamic behavior of hematopoietic stem cells
141. v mice
142. Behavior of hematopoietic stem cells in a large animal
143. Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates
144. + progenitors using clinically feasible methods
145. + cells in Thy-1 expression and cell cycle status in nonhuman primates mobilized or not mobilized with granulocyte colony-stimulating factor and/or stem cell factor