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Antisense oligonucleotides as therapeutic agents: Is the bullet really magical?
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Stein CA, Cheng YC: Antisense oligonucleotides as therapeutic agents: Is the bullet really magical? Science 1993, 261:1004-1012.
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Stein, C.A.1
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0029608635
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The therapeutic potential of antisense oligodeoxynucleotides
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Sharma HW, Narayanan R: The therapeutic potential of antisense oligodeoxynucleotides. Bioessays 1995, 17:1055-1063. A review of the current status of antisense, from the laboratory to the clinic. Nonspecificity of antisense is discussed in detail.
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Bioessays
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Sharma, H.W.1
Narayanan, R.2
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Specific regulation of gene expression by antisense, sense and antigene nucleic acids
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Helene C, Toulme JJ: Specific regulation of gene expression by antisense, sense and antigene nucleic acids. Biochim Biophys Acta 1990, 1049:99-125.
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A single-injection protein kinase A- directed antisense treatment to inhibit tumour growth
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Nesterova M, Cho-Chung YW: A single-injection protein kinase A- directed antisense treatment to inhibit tumour growth. Nature Med 1995, 1:528-533. A single injection of the antisense ODNs caused pronounced tumor inhibition in vivo. The growth inhibition persisted, even after RIα inhibition was lost, so long as PKA-1 (the RIα-containing holoenzyme) downregulation was present. Results suggest that certain antisense ODNs may require relatively infrequent repetitive dosing for in vivo efficacy.
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Nature Med
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Nesterova, M.1
Cho-Chung, Y.W.2
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6
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0028823757
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In vitro and in vivo inhibition of human papillomavirus type 16 E6 and E7 genes
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Tan TMC, Ting RCY: In vitro and in vivo inhibition of human papillomavirus type 16 E6 and E7 genes. Cancer Res 1995, 55:4599-4605.
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Tan, T.M.C.1
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7
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0029977448
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Antitumor activity of a phosphorothioate antisense oligodeoxynucleotide targeted against c-raf kinase
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50 0.06-0.6 mg/kg) of the antisense PS ODN was seen in these studies. Following a single intravenous administration (at 6 mg/kg), accumulation of antisense ODN at 10 to 50 μm was seen in tumors. The study seems to suggest that tumors may be more sensitive to c-raf inhibition than normal cells, and that c-raf antisense may be effective in multiple tumors. Detailed studies with tumors containing different ras mutations are needed, however, to clarify the impact of this molecular target.
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Nature Med
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Monia, B.P.1
Johnston, J.F.2
Geiger, T.3
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Fabbro, D.5
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8
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0029360643
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Prevention of tumor formation in a mouse model of Burkitt's lymphoma by 6 weeks of treatment with anti-c-myc DNA phosphorothioate
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Huang Y, Snyder R, Kligshteyn M, Wickstrom E: Prevention of tumor formation in a mouse model of Burkitt's lymphoma by 6 weeks of treatment with anti-c-myc DNA phosphorothioate. Mol Med 1995, 1:647-658.
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Huang, Y.1
Snyder, R.2
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9
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0027093252
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Down-regulation of c-MYC antigen expression in lymphocytes of Emu-c-myc transgenic mice treated with anti-c-myc DNA methylphosphonates
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Wickstrom E, Bacon T, Wickstrom EL: Down-regulation of c-MYC antigen expression in lymphocytes of Emu-c-myc transgenic mice treated with anti-c-myc DNA methylphosphonates. Cancer Res 1992, 52:6741-6745.
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Wickstrom, E.1
Bacon, T.2
Wickstrom, E.L.3
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0030066249
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In vivo inhibition of hepatitis B virus gene expression by antisense phosphorothioate oligonucleotides
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Moriya K, Matsukura M, Kurokawa K, Koike K: In vivo inhibition of hepatitis B virus gene expression by antisense phosphorothioate oligonucleotides. Biochem Biophys Res Commun 1996, 218:217-223.
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Biochem Biophys Res Commun
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Moriya, K.1
Matsukura, M.2
Kurokawa, K.3
Koike, K.4
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11
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0028785540
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Leukemia treatment in severe combined immunodeficieny mice by antisense oligodeoxynucleotides targeting cooperating oncogenes
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Skorski T, Nieborowska-Skorska M, Campbell K, Iozzo RV, Zon G, Darzynkiewicz Z, Calabretta B: Leukemia treatment in severe combined immunodeficieny mice by antisense oligodeoxynucleotides targeting cooperating oncogenes. J Exp Med 1995, 182:1645-1653. Cancer is seen together with multiple genetic defects. Single-gene targeting may not be efficacious owing to alternative pathways. This report demonstrates the potential of targeting multiple genes in cancer therapy. The use of multiple antisense ODNs can, however, increase the complexity of interpretations.
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J Exp Med
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Skorski, T.1
Nieborowska-Skorska, M.2
Campbell, K.3
Iozzo, R.V.4
Zon, G.5
Darzynkiewicz, Z.6
Calabretta, B.7
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12
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0027319530
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Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells
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Yaswen P, Stampfer MR, Ghosh K, Cohen JS: Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells. Antisense Res Dev 1993, 3:67-77.
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Yaswen, P.1
Stampfer, M.R.2
Ghosh, K.3
Cohen, J.S.4
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13
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0028924169
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Sequence context of antisense RelA/NF-κB phosphorothioates determines specificity
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Maltese JY, Vassilev L, Sharma HW, Narayanan R: Sequence context of antisense RelA/NF-κB phosphorothioates determines specificity. Nucleic Acids Res 1995, 7:1146-1151. A set of contiguous dG residues in an ODN (ie, a G quartet, 4 Gs) is believed to contribute to undefined nonspecificity. A systematic analysis of refA antisense PS ODN, which contains a G quartet, was made by designing more than 40 ODNs with the G quartet in varying positions. Results indicate that generalization with reference to the presence of a G quartet in an ODN cannot be made. The context, ie, the sequences that surround the G quartet, is the critical factor for the G quartet to exert its effect.
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Nucleic Acids Res
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Maltese, J.Y.1
Vassilev, L.2
Sharma, H.W.3
Narayanan, R.4
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14
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0028176489
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Aptameric inhibition of p210 bcr-abl tyrosine kinase autophosphorylation by oligodeoxynucleotides of defined sequence and backbone structure
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Bergan R, Connell Y, Fahmy B, Kyle E, Neckers L: Aptameric inhibition of p210 bcr-abl tyrosine kinase autophosphorylation by oligodeoxynucleotides of defined sequence and backbone structure. Nucleic Acids Res 1994, 22:2150-2154.
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Bergan, R.1
Connell, Y.2
Fahmy, B.3
Kyle, E.4
Neckers, L.5
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15
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0029007784
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Antisense inhibition of urokinase reduces spread of human ovarian cancer in mice
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Wilhelm O, Schmitt M, Hohl S, Senekowitsch R, Graeff H: Antisense inhibition of urokinase reduces spread of human ovarian cancer in mice. Clin Exp Metastasis 1995, 13:296-302.
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Clin Exp Metastasis
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Wilhelm, O.1
Schmitt, M.2
Hohl, S.3
Senekowitsch, R.4
Graeff, H.5
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16
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0029031549
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A gene therapy strategy using a transcription factor decoy of the E2F binding site inhibits smooth muscle proliferation in vivo
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Morishita R, Gibbons GH, Horiuchi M, Ellison KE, Nakajima M, Zhang L, Kaneda Y, Ogihara T, Dzau VJ: A gene therapy strategy using a transcription factor decoy of the E2F binding site inhibits smooth muscle proliferation in vivo. Proc Natl Acad Sci U S A 1995, 92:5855-5859. Transcription factors bind to a distinct set of DNA sequences with high affinity. This report demonstrates that transcription factor function in vivo can be blocked by a double-stranded PS ODN (transcription factor decoy [TFD]), which acts as a competitor. This is the first report of the use of a TFD in a preclinical model. While nonspecific components of PS ODNs may still play a part, the TFD approach provides a nonantisense mechanism for downregulation of transcription factor function. For details of the TFD approach, see Sharma et al. (Anticancer Res 1996, 16:61-70).
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(1995)
Proc Natl Acad Sci U S A
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, pp. 5855-5859
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Morishita, R.1
Gibbons, G.H.2
Horiuchi, M.3
Ellison, K.E.4
Nakajima, M.5
Zhang, L.6
Kaneda, Y.7
Ogihara, T.8
Dzau, V.J.9
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17
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0029617368
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A DNA motif present in αV integrin promoter exhibits dual binding preference to distinct transcription factors
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Sharma HW, Higgins-Sochaski KA, Perez JR, Narayanan R: A DNA motif present in αV integrin promoter exhibits dual binding preference to distinct transcription factors. Anticancer Res 1995, 15:1857-1868.
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(1995)
Anticancer Res
, vol.15
, pp. 1857-1868
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Sharma, H.W.1
Higgins-Sochaski, K.A.2
Perez, J.R.3
Narayanan, R.4
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18
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0028946614
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Transcriptional inhibition of the inducible nitric oxide synthese gene by competitive binding of NF-κ/Rel proteins
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Goldring CE, Narayanan R, Lagadec P, Jeannin JF: Transcriptional inhibition of the inducible nitric oxide synthese gene by competitive binding of NF-κ/Rel proteins. Biochem Biophys Res Commun 1995, 209:73-79.
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(1995)
Biochem Biophys Res Commun
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, pp. 73-79
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Goldring, C.E.1
Narayanan, R.2
Lagadec, P.3
Jeannin, J.F.4
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19
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0028785523
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Thiol agents and Bcl-2 identify an alphavirus-induced apoptotic pathway that requires activation of the transcription factor NF-kappa B
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Lin KI, Lee SH, Narayanan R, Baraban JM, Hardwick JM, Ratan RR: Thiol agents and Bcl-2 identify an alphavirus-induced apoptotic pathway that requires activation of the transcription factor NF-kappa B. J Cell Biol 1995, 131:1149-1161.
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J Cell Biol
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Lin, K.I.1
Lee, S.H.2
Narayanan, R.3
Baraban, J.M.4
Hardwick, J.M.5
Ratan, R.R.6
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20
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0029980617
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Transcription factor decoy approach to decipher the role of NF-κB in oncogenesis
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Sharma HW, Perez JR, Higgins-Sochaski KA, Hsiao R, Narayanan R: Transcription factor decoy approach to decipher the role of NF-κB in oncogenesis. Anticancer Res 1996, 16:61-70. This report details the basis of the TFD approach to inhibiting transcription factor function. For a ubiquitous transcription factor such as NF-κB, homo- or heterodimers can be selectively targeted by the TFD approach.
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(1996)
Anticancer Res
, vol.16
, pp. 61-70
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Sharma, H.W.1
Perez, J.R.2
Higgins-Sochaski, K.A.3
Hsiao, R.4
Narayanan, R.5
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21
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0029145127
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Antisense DNA downregulation of the ERBB2 oncogene measured by a flow cytometric assay
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Vaughn JP, Iglehart JD, Demirdji S, Davis P, Babiss LE, Caruthers MH, Marks JR: Antisense DNA downregulation of the ERBB2 oncogene measured by a flow cytometric assay. Proc Natl Acad Sci U S A 1995, 92:8336-8342. This report describes a novel approach to examining antisense effects that allows simultaneous measurement of antisense dose and gene-specific regulation on a per-cell basis. An antisense PS ODN to erB2 was codelivered with a fluorescein isothiocyanate-labeled ODN, and two-color flow cytometry was used to trace the fate of the ODNs. Results support previous contentions that only within a narrow dose range does an antisense PS ODN exhibit activity.
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(1995)
Proc Natl Acad Sci U S A
, vol.92
, pp. 8336-8342
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Vaughn, J.P.1
Iglehart, J.D.2
Demirdji, S.3
Davis, P.4
Babiss, L.E.5
Caruthers, M.H.6
Marks, J.R.7
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22
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0029088680
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De novo decorin gene expression suppresses the malignant phenotype in human colon cancer cells
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Santra M, Skorski T, Calabretta B, Lattime EC, Iozzo RV: De novo decorin gene expression suppresses the malignant phenotype in human colon cancer cells. Proc Natl Acad Sci U S A 1995, 92:7016-7020.
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Proc Natl Acad Sci U S A
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Santra, M.1
Skorski, T.2
Calabretta, B.3
Lattime, E.C.4
Iozzo, R.V.5
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23
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0029130619
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Inhibition of colon cancer cell proliferation by antisense oligonucleotides targeting the messenger RNA of the Ki-ras gene
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Sakakura C, Hagiwara A, Tsujimoto H, Ozaki K, Sakakibara T, Oyama T, Ogaki M, Imanishi T, Yamazaki J, Takahashi T: Inhibition of colon cancer cell proliferation by antisense oligonucleotides targeting the messenger RNA of the Ki-ras gene. Anticancer Drugs 1995, 6:553-561.
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Anticancer Drugs
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Sakakura, C.1
Hagiwara, A.2
Tsujimoto, H.3
Ozaki, K.4
Sakakibara, T.5
Oyama, T.6
Ogaki, M.7
Imanishi, T.8
Yamazaki, J.9
Takahashi, T.10
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24
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0030071190
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Reduction of expression of the multidrug resistance protein (MRP) in human tumor cells by antisense phosphorothioate oligonucleotides
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Stewart AJ, CanitRot Y, Baracchini E, Dean NM, Deeley RG, Cole SPC: Reduction of expression of the multidrug resistance protein (MRP) in human tumor cells by antisense phosphorothioate oligonucleotides. Biochem Pharmacol 1996, 51:461-469.
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Biochem Pharmacol
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Stewart, A.J.1
CanitRot, Y.2
Baracchini, E.3
Dean, N.M.4
Deeley, R.G.5
Cole, S.P.C.6
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25
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0029618209
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Inhibition of chronic myelogenous leukemia cells harboring a BCR-ABL B3A2 junction by antisense oligonucleotides targeted at the B2A2 junction
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Mahon FX, Ripoche J, Pigeonnier V, Jazwiec B, Pigneux A, Moreau JF, Reiffers J: Inhibition of chronic myelogenous leukemia cells harboring a BCR-ABL B3A2 junction by antisense oligonucleotides targeted at the B2A2 junction. Exp Hematol 1995, 23:1606-1611.
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Exp Hematol
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Mahon, F.X.1
Ripoche, J.2
Pigeonnier, V.3
Jazwiec, B.4
Pigneux, A.5
Moreau, J.F.6
Reiffers, J.7
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26
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0028920301
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Effect of IL-6 receptor antisense oligodeoxynucleotide on in vitro proliferation of myeloma cells
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Keller ET, Ershler WB: Effect of IL-6 receptor antisense oligodeoxynucleotide on in vitro proliferation of myeloma cells. J Immunol 1995, 154:4091-4098.
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J Immunol
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Keller, E.T.1
Ershler, W.B.2
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27
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0030049639
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Tumour cells surviving in vivo cisplatin chemotherapy display elevated c-myc expression
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Walker TL, White JD, Esdale WJ, Burton MA, Decruz EE: Tumour cells surviving in vivo cisplatin chemotherapy display elevated c-myc expression. Br J Cancer 1996, 73:610-614.
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Br J Cancer
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Walker, T.L.1
White, J.D.2
Esdale, W.J.3
Burton, M.A.4
Decruz, E.E.5
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28
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0029092545
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Amphiregulin antisense oligodeoxynucleotides inhibit growth and transformation of a human colon carcinoma cell line
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Normanno N, Selvam MP, Bianco C, Damiano V, De Angelis E, Grassi M, Magliulo G, Tortora G, Salomon DS, Ciardiello F: Amphiregulin antisense oligodeoxynucleotides inhibit growth and transformation of a human colon carcinoma cell line. Int J Cancer 1995, 62:762-766.
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Int J Cancer
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Normanno, N.1
Selvam, M.P.2
Bianco, C.3
Damiano, V.4
De Angelis, E.5
Grassi, M.6
Magliulo, G.7
Tortora, G.8
Salomon, D.S.9
Ciardiello, F.10
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29
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0029366403
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Antisense oligodeoxyribonucleotide down-regulation of bcl-2 gene expression inhibits growth of the low-grade non-Hodgkin's lymphoma cell line WSU-FSCCL
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Smith MR, Abubakr Y, Mohammad R, Xie T, Hamdan M, Al-Katib A: Antisense oligodeoxyribonucleotide down-regulation of bcl-2 gene expression inhibits growth of the low-grade non-Hodgkin's lymphoma cell line WSU-FSCCL. Cancer Gene Ther 1995, 2:207-212.
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Cancer Gene Ther
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Smith, M.R.1
Abubakr, Y.2
Mohammad, R.3
Xie, T.4
Hamdan, M.5
Al-Katib, A.6
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30
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0029870737
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Stromal cells regulate bcl-2 and bax expression in pro-B cells
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Gibson LF, Piktel D, Narayanan R, Nunez G, Landreth KS: Stromal cells regulate bcl-2 and bax expression in pro-B cells. Exp Hematol 1996, 24:628-637.
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Exp Hematol
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Gibson, L.F.1
Piktel, D.2
Narayanan, R.3
Nunez, G.4
Landreth, K.S.5
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31
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0028904465
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Inhibition of proliferation by L-myc antisense DNA for the translational initiation site in human small cell lung cancer
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Dosaka-Akita H, Akie K, Hiroumi H, Kinoshila I, Kawakami Y, Murakami A: Inhibition of proliferation by L-myc antisense DNA for the translational initiation site in human small cell lung cancer. Cancer Res 1995, 55:1559-1564.
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Cancer Res
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Dosaka-Akita, H.1
Akie, K.2
Hiroumi, H.3
Kinoshila, I.4
Kawakami, Y.5
Murakami, A.6
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32
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0030031660
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Antisense inhibition of parathyroid hormone related peptide gene expression reduces malignant pituitary tumor progression and metastases in the rat
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Akino K, Ohtsuru A, Yano H, Ozeki S, Namba H, Nakashima M, Ito M, Matsumoto T, Yamashita S: Antisense inhibition of parathyroid hormone related peptide gene expression reduces malignant pituitary tumor progression and metastases in the rat Cancer Res 1996, 56:77-86.
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Cancer Res
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Akino, K.1
Ohtsuru, A.2
Yano, H.3
Ozeki, S.4
Namba, H.5
Nakashima, M.6
Ito, M.7
Matsumoto, T.8
Yamashita, S.9
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33
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0028964323
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Expression of antisense to DNA methyltransferase mRNA induces DNA demethylation and inhibits tumorigenesis
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Macleod AR, Szyf M: Expression of antisense to DNA methyltransferase mRNA induces DNA demethylation and inhibits tumorigenesis. J Biol Chem 1995, 270:8037-8043.
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J Biol Chem
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Macleod, A.R.1
Szyf, M.2
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34
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0029865486
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Eradication of established intracranial rat gliomas by transforming growth factor β antisense gene therapy
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Fakhrai H, Dorigo O, Shawler DL, Lin H, Mercola D, Black KL, Royston I, Sobol RE: Eradication of established intracranial rat gliomas by transforming growth factor β antisense gene therapy. Proc Natl Acad Sci U S A 1996, 93:2909-2914.
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Proc Natl Acad Sci U S A
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Fakhrai, H.1
Dorigo, O.2
Shawler, D.L.3
Lin, H.4
Mercola, D.5
Black, K.L.6
Royston, I.7
Sobol, R.E.8
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35
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0028820153
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Transforming growth factor β1 (TGFβ1) is an autocrine positive regulator of colon carcinoma U9 cells in vivo as shown by transfection of a TGFβ1 antisense expression plasmid
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Huang F, Newman E, Theodorescu D, Kerbel RS, Friedman E: Transforming growth factor β1 (TGFβ1) is an autocrine positive regulator of colon carcinoma U9 cells in vivo as shown by transfection of a TGFβ1 antisense expression plasmid. Cell Growth Differ 1995, 6:1635-1642.
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Cell Growth Differ
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Huang, F.1
Newman, E.2
Theodorescu, D.3
Kerbel, R.S.4
Friedman, E.5
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36
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0030029271
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Inhibition of growth of C6 glioma cells in vivo by expression of antisense vascular endothelial growth factor sequence
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Saleh M, Stacker SA, Wilks AF: Inhibition of growth of C6 glioma cells in vivo by expression of antisense vascular endothelial growth factor sequence. Cancer Res 1996, 56:393-401.
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Cancer Res
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Saleh, M.1
Stacker, S.A.2
Wilks, A.F.3
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37
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0030045221
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Tissue-targeted antisense c-fos retroviral vector inhibits established breast cancer xenografts in nude mice
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Arteaga CL, Holt JT: Tissue-targeted antisense c-fos retroviral vector inhibits established breast cancer xenografts in nude mice. Cancer Res 1996, 56:1098-1103. Inhibition of c-fos oncogene mediated by a retroviral vector was achieved in a tissue-specific manner (breast). Growth and invasion of MCF-7 breast carcinoma was inhibited by in vivo injection of the retroviral antisense. This study provides a rationale for targeted gene therapy in breast cancer.
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(1996)
Cancer Res
, vol.56
, pp. 1098-1103
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Arteaga, C.L.1
Holt, J.T.2
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38
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0001457668
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Amplification of AKT2 in human pancreatic cancer cells and inhibition of AKT2 expression and tumorigenicitiy by antisense RNA
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Cheng JQ, Ruggeri B, Klein WM, Sonada G, Altomare DA, Watson DK, Testa JR: Amplification of AKT2 in human pancreatic cancer cells and inhibition of AKT2 expression and tumorigenicitiy by antisense RNA. Proc Natl Acad Sci U S A 1996, 93:3636-3641.
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Proc Natl Acad Sci U S A
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Cheng, J.Q.1
Ruggeri, B.2
Klein, W.M.3
Sonada, G.4
Altomare, D.A.5
Watson, D.K.6
Testa, J.R.7
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39
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0029080861
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Antisense to cyclin D1 inhibits growth and reverses the transformed phenotype of human esophageal cancer cells
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Zhou P, Jiang W, Zhang Y, Kahn SM, Schieren I, Santella RM, Weinstein IB: Antisense to cyclin D1 inhibits growth and reverses the transformed phenotype of human esophageal cancer cells. Oncogene 1995, 11:571-580.
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Oncogene
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Zhou, P.1
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Weinstein, I.B.7
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40
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0029125689
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Growth inhibition of MCF-7 breast cancer cells by stable expression of an insulin-like growth factor I receptor antisense ribonucleic acid
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Neuenschwander S, Roberts CT JR, Leroith D: Growth inhibition of MCF-7 breast cancer cells by stable expression of an insulin-like growth factor I receptor antisense ribonucleic acid. Endocrinology 1995, 136:4298-4303.
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Endocrinology
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Neuenschwander, S.1
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Leroith, D.3
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41
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0028889597
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Inhibition of fibroblast growth factor 2 expression by antisense RNA induced a loss of the transformed phenotype in a human hepatoma cell line
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Maret A, Galy B, Arnaud E, Bayard F, Prats H: Inhibition of fibroblast growth factor 2 expression by antisense RNA induced a loss of the transformed phenotype in a human hepatoma cell line. Cancer Res 1995, 55:5075-5079.
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Modulation of proliferation and tumorigenic potential of cervical cercinoma cells by the expression of sense and antisense p53
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Velasco JA, Stevens CW, Esteban JM, Ruthsatz MK, Ramsamooj P, Dritschilo A, Notario V: Modulation of proliferation and tumorigenic potential of cervical cercinoma cells by the expression of sense and antisense p53. Int J Oncol 1995, 7:883-888.
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Dritschilo, A.6
Notario, V.7
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43
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0029000974
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Expression of antisense epidermal growth factor receptor RNA downmodulates the malignant behavior of human colon cancer cells
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Chakrabarty S, Rajagopal S, Huang S: Expression of antisense epidermal growth factor receptor RNA downmodulates the malignant behavior of human colon cancer cells. Clin Exp Metastasis 1995, 13:191-195.
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44
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0029124127
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Liposome-mediated in vivo gene transfer of antisense K-ras construct inhibits pancreatic tumor dissemination in the murine peritoneal cavity
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Aoki K, Yoshida T, Sugimura T, Terada M: Liposome-mediated in vivo gene transfer of antisense K-ras construct inhibits pancreatic tumor dissemination in the murine peritoneal cavity. Cancer Res 1995, 55:3810-3816. This study documents the use of liposomes to deliver an antisense construct into the peritoneum of nude mice bearing tumors. Injected DNA was detected in multiple tissues (except brain) at day 24. No treatment-related toxicity was seen. This method may offer an effective alternative to viral vectors for gene therapy.
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Cancer Res
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Aoki, K.1
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Feng M, Cabrera G, Deshane J, Scanlon KJ, Curiel DT: Neoplastic reversion accomplished by high efficiency adenoviral-mediated delivery of an anti-ras ribozyme. Cancer Res 1995, 55:2024-2028. This is the first demonstration of an adenovirus-based vector for delivery of a ribozyme construct in mice.
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Cancer Res
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Feng, M.1
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46
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0029096515
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The RNA component of human telomerase
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Feng J, Funk WD, Wang SS, Weinrich SL, Avilion AA, Chiu CP, Adams RR, Chang E, Allsopp RC, Yu J, Le S, West MD, Harley CB, Andrews WH, Greider GW, Villeponteau B: The RNA component of human telomerase. Science 1995, 269:1236-1241.
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47
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Akhtar S, ed: Delivery Strategies for Antisense Oligonucleotide Therapeutics. Boca Raton, FI: CRC Press, Inc.; 1995. This comprehensive volume reviews the major hurdles to oligonucleotide delivery and discusses many of the current strategies being developed for the improved delivery and targeting of oligonucleotides to desired sites of the body.
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Delivery Strategies for Antisense Oligonucleotide Therapeutics
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Akhtar, S.1
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48
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49
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Improved cellular delivery of antisense oligonucleotides using transferrin receptor antibody-oligonucleotide conjugates
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Walker I, Irwin WJ, Akhtar S: Improved cellular delivery of antisense oligonucleotides using transferrin receptor antibody-oligonucleotide conjugates. Pharm Res 1995, 12:1548-1553. Transferrin receptor antibodies have been used in the past to deliver peptides and small-molecular-weight compounds. This report documents the delivery of ODNs by coupling to the antibody. It is conceivable that such coupling may enable the delivery of ODNs across the blood-brain barrier, since the microvessel endothelial cells in the barrier have an abundance of the transferrin receptor.
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Pharm Res
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Walker, I.1
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50
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Biodegradable poly(L-lactic acid) matrices for the sustained delivery of antisense oligonucleotides
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Lewis KJ, Irwin WJ, Akhtar S: Biodegradable poly(L-lactic acid) matrices for the sustained delivery of antisense oligonucleotides. J Controlled Rel 1996, 37:173-183. Uptake of ODNs by cells is poor; nontoxic delivery systems are crucial to efficacy. This preliminary report demonstrates the potential utility of a biodegradable poly(L-lactic acid) matrix in achieving sustained release of ODNs in cell-free systems.
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J Controlled Rel
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Lewis, K.J.1
Irwin, W.J.2
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51
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0028924955
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Delivery of antisense oligodeoxyribonucleotides against the human epidermal growth factor receptor into cultured KB cells with liposomes conjugated to folate polyethylene glycol
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Wang S, Lee RJ, Cauchon G, Gorenstein DG, Low PS: Delivery of antisense oligodeoxyribonucleotides against the human epidermal growth factor receptor into cultured KB cells with liposomes conjugated to folate polyethylene glycol. Proc Natl Acad Sci U S A 1995, 92:3318-3322.
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Wang, S.1
Lee, R.J.2
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Gorenstein, D.G.4
Low, P.S.5
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52
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0029872147
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A serum-resistant cytofectin for cellular delivery of antisense oligodeoxynucleotides and plasmid DNA
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Lewis JG, Lin KY, Kothavale A, Flanagan WM, Matteucci MD, Deprince RB, Mook RA JR, Hendren RW, Wagner RW: A serum-resistant cytofectin for cellular delivery of antisense oligodeoxynucleotides and plasmid DNA. Proc Natl Acad Sci U S A 1996, 93:3176-3181. This report describes development of a novel lipofectamine that represents a major improvement over existing reagents for ODN delivery. This formulation delivers ODNs and plasmids into many (but not all) cell types. Although the mechanism is not clear, it appears to destabilize the endosomal membranes, permitting entry of ODNs into the cytoplasm. Once in the nucleus, the ODNs are not diffused back into the cytoplasm. This formulation enabled the use of nanomolar concentrations of PS ODNs and may thus circumvent the nonspecificity seen with these ODNs at high concentrations.
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Proc Natl Acad Sci U S A
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Lewis, J.G.1
Lin, K.Y.2
Kothavale, A.3
Flanagan, W.M.4
Matteucci, M.D.5
Deprince, R.B.6
Mook Jr., R.A.7
Hendren, R.W.8
Wagner, R.W.9
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53
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0029127845
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Pharmacokinetics of an anti-HIV antisense oligodeoxynucleotide phosphorothioate (GEM 91) in HIV-infected subjects
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Zhang R, Yan J, Shahinian H, Amin G, Lu Z, Liu T, Saag MS, Jiang Z, Temsamani J, Martin RR, Schechter PJ, Agrawal S, Diasio RB: Pharmacokinetics of an anti-HIV antisense oligodeoxynucleotide phosphorothioate (GEM 91) in HIV-infected subjects. Clin Pharmacol Ther 1995, 58:44-53.
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Zhang, R.1
Yan, J.2
Shahinian, H.3
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Lu, Z.5
Liu, T.6
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Jiang, Z.8
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Martin, R.R.10
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54
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Pharmacokinetics of antisense oligonucleotides
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Agrawal S, Temsamani J, Galbraith W, Tang J: Pharmacokinetics of antisense oligonucleotides. Clin Pharmacokinet 1995, 28:7-16. A comprehensive pharmacokinetics study of PO, PS, and MP ODNs in mice, rats, monkeys, and humans.
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Clin Pharmacokinet
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Agrawal, S.1
Temsamani, J.2
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55
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0029100397
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Adsorption, tissue distribution and in vivo stability in rats of a hybrid antisense oligonucleotide following oral administration
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Agrawal S, Zhang X, Lu Z, Zhao H, Tamburin JM, Yan J, Cai H, Diasio RB, Habus I, Jiang Z, Iyer RP, Yu D, Zhang R: Adsorption, tissue distribution and in vivo stability in rats of a hybrid antisense oligonucleotide following oral administration. Biochem Pharmacol 1995, 50:571-576. The ability to administer ODNs orally would greatly simplify their therapeutic use. This is the first report of such delivery. Hybrid ODNs were shown to be absorbed intact through the gastrointestinal tract, a finding that may be important in the development of antisense ODNs as therapeutic agents.
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Biochem Pharmacol
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Agrawal, S.1
Zhang, X.2
Lu, Z.3
Zhao, H.4
Tamburin, J.M.5
Yan, J.6
Cai, H.7
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Jiang, Z.10
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Zhang, R.13
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56
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9244223546
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Phase I trial of an antisense oligonucleotide OL(1)p53 in hematologic malignancies
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Bishop MR, Iversen PL, Bayever E, Sharp JG, Greiner TC, Copple BL, Ruddon R, Zon G, Spinolo J, Arneson M, Armitage JO, Kessinger A: Phase I trial of an antisense oligonucleotide OL(1)p53 in hematologic malignancies. J Clin Oncol 1996, 14:1320-1326.
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Bishop, M.R.1
Iversen, P.L.2
Bayever, E.3
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Greiner, T.C.5
Copple, B.L.6
Ruddon, R.7
Zon, G.8
Spinolo, J.9
Arneson, M.10
Armitage, J.O.11
Kessinger, A.12
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57
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0026573769
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Phosphorothioate oligonucleotides are inhibitors of human DNA polymerases and RNase H: Implications for antisense technology
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Gao WY, Han FS, Storm C, Egan W, Cheng YC: Phosphorothioate oligonucleotides are inhibitors of human DNA polymerases and RNase H: Implications for antisense technology. Mol Pharmacol 1992, 41:223-229.
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Mol Pharmacol
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Gao, W.Y.1
Han, F.S.2
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58
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0026969188
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Template, phosphorothioate oligonucleotides duplexes as inhibitors of HIV-1 reverse transcriptase
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Maury G, Abdelaziz E, Morvan F, Muller B, Imbach J, Goody RS: Template, phosphorothioate oligonucleotides duplexes as inhibitors of HIV-1 reverse transcriptase. Biochem Biophys Res Commun 1992, 186:1249-1256.
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Biochem Biophys Res Commun
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59
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0026058794
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Phosphorothioate oligodeoxycytidine interferes with binding of HIV-1 and HIV-2 in vitro
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60
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0029975255
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Telomerase as a potential molecular target to study G-quartet phosphorothioates
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Sharma HW, Hsiao R, Narayanan R: Telomerase as a potential molecular target to study G-quartet phosphorothioates. Antisense Res Dev 1996, 6:3-7. This is yet another example of the nonspecific effects of G-quartet containing PS ODNs. Depending on the context of their occurrence, G-quartet PS ODNs inhibited telomerase (an enzyme implicated in cell senescence and immortalization) activity in vitro. Phosphodiester and MP ODNs had no effect.
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Antisense Res Dev
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Sharma, H.W.1
Hsiao, R.2
Narayanan, R.3
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61
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0028114548
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Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding
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Brown DA, Kang SH, Gryaznov SM, Dedionisio L, Heidenreich O, Sullivan S, Xu X, Nerenberg MI: Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding. J Biol Chem 1994, 269:26801-26805.
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Brown, D.A.1
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Sullivan, S.6
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62
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0028340194
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Sequence independent induction of Sp-1 transcription factor by phosphorothioate oligodeoxynucleotides
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Perez JR, Li Y, Stein CA, Majumder S, Van Oorschot A, Narayanan R: Sequence independent induction of Sp-1 transcription factor by phosphorothioate oligodeoxynucleotides. Proc Natl Acad Sci U S A 1994, 91:5957-5961.
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Narayanan, R.6
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63
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0027163032
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Immune stimulation by an antisense oligomer complementary to the rev gene of HIV-I
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Branda RF, Moore AL, Mathews L, McCormack JJ, Zon G: Immune stimulation by an antisense oligomer complementary to the rev gene of HIV-I. Biochem Pharmacol 1993, 45:2037-2043.
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Biochem Pharmacol
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64
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0027762537
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A sense phosphorothioate oligonucleotide directed to the Initiation codon of transcription factor NF-κB p65 causes sequence-specific immune stimulation
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McIntyre KW, Lombard-Gillooly K, Perez JR, Kunsch C, Sarmiento UM, Larigan JD, Landreth KT, Narayanan R: A sense phosphorothioate oligonucleotide directed to the Initiation codon of transcription factor NF-κB p65 causes sequence-specific immune stimulation. Antisense Res Dev 1993, 3:309-322.
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Antisense Res Dev
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McIntyre, K.W.1
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Larigan, J.D.6
Landreth, K.T.7
Narayanan, R.8
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65
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0028931102
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CpG motifs in bacterial DNA trigger direct B-cell activation
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Krieg AM, Yi AK, Matson S, Waldschmidt TJ, Bishop GA, Teasdale R, Koretzky GA, Klinman DM: CpG motifs in bacterial DNA trigger direct B-cell activation. Nature 1995, 374:546-549.
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Nature
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66
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Multiple mechanisms may contribute to the cellular anti-adhesive effects of phosphorothioate oligodeoxynucleotides
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Khaled, Z.1
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Stein, C.A.7
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