Targeted gene modification for gene therapy of stem cells

The International Journal of Cell Cloning

Volumn 8, Issue 2, 1990, Pages 80-95

  Boggs, Sallie S ^a  

^a UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

Author keywords

Gene modification; Gene therapy; Genetic modification; Hematopoietic stem cells; Homologous recombination

Indexed keywords

CELL CULTURE; GENE THERAPY; GENETIC ENGINEERING; GENETIC RECOMBINATION; HEMATOPOIETIC STEM CELL; REVIEW;

ANIMAL; DNA, RECOMBINANT; EUKARYOTIC CELLS; GENE THERAPY; GENETIC DISEASES, INBORN; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMAN; MICE; MICE, TRANSGENIC; MICROINJECTIONS; RECOMBINATION, GENETIC; RETROVIRIDAE; TRANSFECTION; TRANSFORMATION, GENETIC;

EID: 0025312204     PISSN: 07371454     EISSN: 15494918     Source Type: Journal    
DOI: 10.1002/stem.5530080202     Document Type: Article

References (84)

1. Gene transfer in intact animals
2. Genetic transformation of murine bone marrow cells to methotrexate resistance
3. Electroporation for the efficient transfection of mammalian cells with DNA
4. Introduction and expression of DNA molecules in eukaryotic cells by electroporation
5. Efficient transformation and frequent single‐site, single‐copy insertion of DNA can be obtained in mouse erythroleukemia cells transformed by electroporation
6. Insertion of DNA sequences into the human chromosomal beta‐globin locus by homologous recombination
7. Short‐term efficient expression of transfected DNA in human hematopoietic cells by electroporation: definition of parameters and use of chemical stimulators
8. Optimizing electroporation parameters for a variety of human hematopoietic cell lines
9. In vivo expression of a nonselected gene transferred into murine hematopoietic stem cells by electroporation
10. Use of electroporation for high‐molecular weight DNA‐mediated gene transfer
11. Developmental potential and dynamic behavior of hematopoietic stem cells
12. Progress toward human gene therapy
13. Gene transfer into hemopoietic stem cells using retroviral vectors
14. Retrovirus‐mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo
15. Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse
16. Restriction of expression of an integrated recombinant retrovirus in primary but not immortalized murine hematopoietic stem cells
17. Design of retrovirus vectors for transfer and expression of the human beta‐globin gene
18. Retrovirus‐mediated transfer and expression of drug resistance genes in human haematopoietic progenitor cells
19. Developmental regulation of a cloned adult beta‐globin gene in transgenic mice
20. Erythroid‐specific expression of human beta‐globin genes in transgenic mice
21. Germ‐line transformation of mice
22. The human beta‐globin gene contains a downstream developmental specific enhancer
23. Position‐independent, high‐level expression of the human beta‐globin gene in transgenic mice
24. The complete sequence of dystrophin predicts a rod‐shaped cytoskeletal protein
25. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges
26. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells
27. The somatic cell as a test tube: comparison between various transfer techniques
28. Microinjection of tissue culture cells using glass capillaries: methods
29. Production of chimaeric mice containing embryonic stem (ES) cells carrying a homoeobox Hox 1.1 allele mutated by homologous recombination
30. Purification and characterization of mouse hematopoietic stem cells
31. Isolation of haemopoietic spleen colony forming cells
32. Isolation of murine pluripotent hemopoietic stem cells
33. Targeted modification of human chromosomal genes
34. Yeast transformation: a model system for the study of recombination
35. The double‐strand‐break repair model for recombination
36. Effect of limited homology on gene conversion in a Saccharomyces cerevisiae plasmid recombination system
37. The mechanism of conservative site‐specific recombination
38. Homologous recombination in monkey cells and human cell‐free extracts
39. A DNA‐recombinogenic catalyzed by human cell extracts
40. Homologous recombination catalyzed by mammalian cell extracts
41. Homologous recombination catalyzed by mammalian cell extracts in vitro
42. Sister chromatid exchange formation
43. Gene amplication
44. Genetic demonstration of mitotic recombination in cultured Chinese hamster cell hybrids
45. Nonreciprocal exchanges of information between DNA duplexes coinjected into mammalian cell nuclei
46. Somatic generation of antibody diversity
47. Specific chromosomal translocations and the genesis of B‐cell‐derived tumors in mice and men
48. Analysis of homologous recombination in cultured mammalian cells
49. Analysis of homologous recombination in cultured mammalian cells in transient expression and stable transformation assays
50. Homologous recombination between plasmids in mammalian cells can be enhanced by treatment of input DNA
51. Patterns of integration of DNA microinjected into cultured mammalian cells: evidence for homologous recombination between injected plasmid DNA molecules
52. Model for homologous recombination during transfer of DNA into mouse L cells: role for DNA ends in the recombination process
53. Effect of insertions, deletions, and double‐strand breaks on homologous recombination in mouse L cells
54. Rapid assay for extrachromosomal homologous recombination in monkey cells
55. Double‐strand gap repair results in homologous recombination in mouse L cells
56. Site‐directed mutagenesis by gene targeting in mouse embryo‐derived stem cells
57. The minimum amount of homology required for homologous recombination in mammalian cells
58. DNA sequence homology and chromosomal deletion at a site of SV40 DNA integration
59. Homologous and nonhomologous recombination in monkey cells
60. High frequency targeting of genes to specific sites in the mammalian genome
61. High frequency of homologous recombination in mammalian cells between endogenous and introduced SV40 genomes
62. Homologous integration in mammalian cells without target gene selection
63. Altering the genome by homologous recombination
64. Disruption of the proto‐oncogene int‐2 in mouse embryo‐derived stem cells: a general strategy for targeting mutations to non‐selectable genes
65. Targeting of nonexpressed genes in embryonic stem cells via homologous recombination
66. Recombination in mouse L cells between DNA introduced into cells and homologous chromosomal sequences
67. Replacement of chromosomal segments with altered DNA sequences constructed in vitro
68. Targetted correction of a mutant HPRT gene in mouse embryonic stem cells
69. Recombinant fragment assay for gene targetting based on the polymerase chain reaction
70. Production of a mutation in mouse En‐2 gene by homologous recombination in embryonic stem cells
71. Recombinant gene expression in vivo within endothelial cells of the arterial wall
72. Partial correction of an inherited biochemical defect of skeletal muscle by grafts of normal muscle precursor cells
73. Bone marrow transplantation for genetic disorders in 1988—a review
74. Experience with bone marrow transplantation for inborn errors of metabolism
75. Displacement bone marrow transplantation and immunoprophylaxis for genetic diseases
76. Bone marrow transplantation for thalassemia: 156 cases transplanted in Pesaro
77. Marrow biology and stem cells
78. The identification in adult bone marrow of pluripotent and restricted stem cells of the myeloid and lymphoid systems
79. Cytological evidence for a relationship between normal hematopoietic colony‐forming cells and cells of the lymphoid system
80. o periods and constant doubling rates of early pluripotent hemopoietic progenitors
81. Defective ability to self‐renew in vitro of highly purified primitive haematopoietic cells
82. Cycling characteristics of endogenous spleen colony‐forming cells as measured with cytosine arabinoside and methotrexate