01. Gene therapy Boulikas.pdf - Gene therapy & Molecular Biology

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Boulikas: An overview on gene therapy Figure 16. Endogenous mammalian β-Gal activity was detected in minced lung preparations from a variety of species following incubation with X-Gal in PBS. Representative fields from paraffin-embedded sections are shown. Original magnification 400X. Airway epithelium, alveolar macrophages and several unidentified cell types are stained blue and are, therefore, positive for β-Gal activity. From Weiss DJ, Liggitt D, Clark JG (1997) In situ histochemical detection of β-galactosidase activity in lung: assessment of X-Gal reagent in distinguishing lacZ gene expression and endogenous β-galactosidase activity. Hum Gene Ther 8, 1545-1554. With the kind permission of the authors (Daniel Weiss, Fred Hutchinson Cancer Research Center) and Mary Ann Liebert, Inc. 42
Gene Therapy and Molecular Biology Vol 1, page 43 Figure 17. Endogenous mammalian β-Gal activity was not detected following incubation with X-Gal at pH>7.5 whereas bacterial β- Gal activity was detected at pH 8.0-8.5 in airway epithelial cells in the transgenic Rosa-26 mouse. Original magnification 250X. From Weiss DJ, Liggitt D, Clark JG (1997) In situ histochemical detection of β-galactosidase activity in lung: assessment of X-Gal reagent in distinguishing lacZ gene expression and endogenous β-galactosidase activity. Hum Gene Ther 8, 1545-1554. With the kind permission of the authors (Daniel Weiss, Fred Hutchinson Cancer Research Center) and Mary Ann Liebert, Inc. Table 2. Reporter gene transfer in vivo Gene delivered CAT and luciferase Luciferase/ CMV construct and lacZ Luciferase/ CMV lacZ and HSV-tk/IL- 2 Human disease Vector Method/Goal Results Reference none DOTM Expression of reporter genes in CMV promoter was most effective; Zhu et al, 1993 A: mouse tissues after a single expression in vascular endothelia, DOPE intravenous injection. extravascular and parenchymal cells present in lung, spleen, and heart; lower expression in all other tissues; persisted for 9 weeks. none Transfe To show effectiveness in gene Transient expression of luciferase in striatal Schwartz et al, ctam transfer after intracranial injection parenchymal cells; lipospermine:DNA 1995 (DOGS of liposome-plasmid complexes charge ratio of 2 or smaller was most :DOPE ) into the newborn mouse. effective in vivo. none Cationi Distribution of luciferase gene Use of viral origins of replication and T Thierry et al, c expression among tissues and antigen cDNA in vector sustained expression 1995 liposo persistence of expression after up to 3 months primarily in the lung in mice. mes systemic injection. glioma AAV Direct injection of tumors induced 30-40% of the cells along the needle track Okada et al,. from human glioma cells into the expressed β-galactosidase; administration of 1996 brains of nude mice. GCV to the HSV-tk/IL-2 treated animals for 6 days, resulted in a 35-fold reduction in the mean volume of tumors compared with 43
Page 1 and 2: Gene Ther Mol Biol Vol 1, 1-172. Ma
Page 3 and 4: I. Introduction Monumental progress
Page 5 and 6: The use of retroviral vectors in hu
Page 7 and 8: displaying the cleavable EGF domain
Page 9 and 10: molecules in the nucleus (Lowe and
Page 11 and 12: sensitive mutations which allow pro
Page 13 and 14: processed as described in panel A s
Page 15 and 16: On the contrary, the payload capaci
Page 17 and 18: in K562 human erythroleukemia cells
Page 19 and 20: defective HSV virus (DeLuca and Sch
Page 21 and 22: complex into the cytosol from the e
Page 23 and 24: Gene Therapy and Molecular Biology
Page 25 and 26: delivered by Sendai virus-liposome
Page 27 and 28: plasmids with the cell surface, tra
Page 29 and 30: infected by adenovirus alone; infec
Page 33 and 34: B. Transcription factor binding sit
Page 35 and 36: A closely related family of ubiquit
Page 37 and 38: cells. I-CreI is a member of this c
Page 41: C. Considerations of chromatin stru
Page 45 and 46: A. The molecular basis of cancer im
Page 47 and 48: fragments, or heat shock proteins c
Page 49 and 50: ecombinant vaccinia virus expressin
Page 51 and 52: HIV-1 envelope DNA construct develo
Page 53 and 54: inding sites A GYYY oriented in opp
Page 57 and 58: Prives, 1994). Whereas the wild-typ
Page 59 and 60: mutant p53 (mutations on p53 are wi
Page 61 and 62: master regulator of the cell cycle
Page 63 and 64: Work from several groups has shown
Page 65 and 66: chromatin condensation, drop in pH,
Page 67 and 68: Figure 23. A pathway leading to the
Page 69 and 70: Excessive necrotic death in cells o
Page 71 and 72: implantation, and similar processes
Page 73 and 74: normal cells in the surroundings. T
Page 79 and 80: A bicistronic retroviral vector (Ha
Page 81 and 82: C. Antisense ras gene transfer for
Page 83 and 84: equal to 6.0 showed S1 hyperreactiv
Page 85 and 86: protein (e.g. Smith et al, 1995; Fo
Page 87 and 88: transduced primary mouse fibroblast
Page 89 and 90: IL-1 - receptor antagonist protein
Page 91 and 92: p53 lung cancer retroviru s MHC cla
Page 93 and 94:
Gene Therapy and Molecular Biology
Page 95 and 96:
designed by immunization with porti
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Isolation of an RNA aptamer that ca
Page 99 and 100:
induction of human apoE in neonate
Page 101 and 102:
atrium in heart, endothelial cells
Page 103 and 104:
which eliminated tumors in small an
Page 105 and 106:
C. Transfer of other genes against
Page 107 and 108:
Page 109 and 110:
significant reduction in neointima
Page 111 and 112:
(Prehn et al, 1996); this implies a
Page 113 and 114:
B. Approaches to gene therapy of RA
Page 115 and 116:
of human immunoglobulin and antigen
Page 117 and 118:
A. Etiology and mechanisms of destr
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Page 121 and 122:
However, CsA also inhibits the acti
Page 123 and 124:
The peptide kinin, after binding to
Page 125 and 126:
intake when administered to adrenal
Page 127 and 128:
Introgen Therapeutics (Austin and H
Page 129 and 130:
When a subfragment of only 513 bp o
Page 131 and 132:
Armentano D, Zabner J, Sacks C, Soo
Page 133 and 134:
Brady HJM, Miles CG, Pennington DJ,
Page 135 and 136:
Chen J, Stickles RJ, Daichendt KA (
Page 137 and 138:
Day ML, Wu S, Basler JW (1993) Pros
Page 139 and 140:
Farmer G, Bargonetti J, Zhu H, Frie
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Giovannangeli C, Perrouault L, Escu
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the neoplastic phenotype by replace
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integrate in a site-specific fashio
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Li R, Waga S, Hannon GJ, Beach D, S
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adiation-induced apoptosis in the g
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Nakaya T, Iwai S, Fujinaga K, Sato
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Polyak K, Xia Y, Zweier JL, Kinzler
Page 155 and 156:
macrophages from simian immunodefic
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intimal hyperplasia in a rat caroti
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Trono D, Feinberg MB, Baltimore D (
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Wattiaux R, Jadot M, Warnier-Pirott
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similar to mammalian interleukin-1
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24. Gene Marking /Infectious Diseas
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Drug 50. Gene Therapy /Phase I /Can
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76. Gene Therapy /Phase I /Cancer /
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99. Gene Therapy /Phase II /Cancer
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120. Gene Therapy /Phase I /Monogen
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Page 179 and 180:
cancer not specified) /Immunotherap
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