Gene expression by positive feedback activation of a cell...

Details Gene expression by positive feedback activation of a cell... Gene expression by positive feedback activation of a cell...

2005-03-15

2005-03-15

Guzo, David (Department: 1636)

Chemistry: molecular biology and microbiology

Vector, per se

C435S455000, C536S024100

Reexamination Certificate

active

06867036

ABSTRACT:
A nucleic acid construct is described which provides cell type-specific expression of a therapeutic transgene. The construct utilizes a cell type-specific promoter which drives expression of the transgene. A positive feedback loop is introduced through the addition of a amplification promoter element operably linked to the therapeutic transgene and by providing, either as part of the same construct, or in a different construct, a transcription activator for activating the amplification promoter element. In one embodiment, the amplification promoter element is a heat shock response element (HSE) and the transcription activator is HSF-1. The construct enables functional targeting of a therapeutic gene while avoiding undesirable effects in non-targeted cells, by combining sufficiently high-level expression to promote a desirable therapeutic outcome with exceptional tissue specificity. A series of promoter elements, constructs, vectors, and therapeutic approaches is presented for gene therapy of tumors such as melanoma and other genetic diseases.

REFERENCES:
patent: 5137805 (1992-08-01), Kingston et al.
patent: 5738985 (1998-04-01), Miles et al.
patent: 5739018 (1998-04-01), Miyanohara et al.
patent: 5756343 (1998-05-01), Wu et al.
patent: 5770581 (1998-06-01), Weichselbaum et al.
patent: 5827685 (1998-10-01), Lindquist
patent: 6034228 (2000-03-01), Norris et al.
patent: 6037329 (2000-03-01), Baird et al.
patent: 20020042127 (2002-04-01), Russel et al.
He et al., Glycogen Synthase Kinase 3B and Extracellular Signal-Regulated Kinase Inactivate Heat Shock Transcription Factor 1 by Facilitating the Disappearance of Transcriptionally Active Granules after Heat Shock; Mol. and Cell Bio, Nov. 1998, p 6624-6633.*
Cao et al., A Safe, Effective in Vivo Gene Therapy for Melanoma Using Tyrosinase Promoter-Driven Cytosine Deaminase Gene, In vivo 13: 181-188 (1999).*
Takeda et al., Functional Analysis of the cDNA Encoding Human Tyrosinase Precursor; Biochemical and Biophysical Research Communications; vol. 162, No. 3 1989, Aug. 15, 1989, pp. 984-990.*
Eck et al., Gene-Based Therapy Chapter V, Goodman & Gilman's The Pharmacological Basis of Therapeutics; pp. 77-101.*
Gura; Systems for Identifying New Drugs Are Often Faulty; www.sciencemag.org; Science; vol. 278, Nov. 7, 1997, p 1041-1042.*
Rozenberg et al., Alternative gene delivery; S.T.P Pharma Sciences 11 (1) 21-30 2001.*
Nishikawa et al., Nonviral Vectors in the New Millennium: Delivery Barriers in Gene Transfer; Human Gene Therapy 12:861-870 (May 20, 2001).*
Balicki et al., Gene Therapy of Human Disease; Medicine 81:69-86, 2002.*
Emiliusen et al. A transcriptional feedback loop for tissue-specific expression of highly cytotoxic genes which incorporates an immunostimulatory component. Gene therapy 8:987-998, 2001.*
Nettelbeck et al. Gene therapy designer promoters for tumour targeting. TIG 16:174-181, 2000.*
Dang et al. Gene therapy and translational cancer research. Clin. Cancer Res. 5:471-474, 1999.*
Verma et al. Gene therapy—promises, problems and prospects. Nature 389:239-242, 1997.*
Sato et al. Enhanced and specific gene expression via tissue-specific production of Cre recombinaase using adenovirus vector. Biochem. Biophys. Res. Commun. 244:455-462, 1998.*
Cao et al., “A safe, effective in vivo gene therapy for melanoma using tyrosinase promoter-driven cytosine deaminase gene,”In Vivo(Greece), 1999, 13(2):181-187, Abstract only.
Mivechi et al., “Stable Overexpression of Human HSF-1 in Murine Cells Suggests Activation Rather Than Expression of HSF-1 to be the Key Regulatory Step in the Heat Shock Gene Expression,”J. Cell. Biochem.,1995, 59:266-280.
Miyazaki et al., “Activation of of Human Multidrug Resistance-1 Gene Promoter in Response to Heat Shock Stress,”Biochem. Biophys. Res. Comm.,1992, 187(2):677-684.
Park et al., “Augmentation of Melanoma-Specific Gene Expression Using a Tandem Melanocyte-Specific Enhancer Results in Increased Cytotoxicity of the Purine Nucleoside Phosphorylase Gene in Melanoma,”Human Gene Therapy,1999, 10:889-898.
Provisional Appl. No. 60/193,977, Filed Mar. 31, 2000.
GenBank Accession No. A23284.
GenBank Accession No. D49357.
GenBank Accession No. D86181.
GenBank Accession No. L12392.
GenBank Accession No. M11147.
GenBank Accession No. M13142.
GenBank Accession No. M14338.
GenBank Accession No. M60091.
GenBank Accession No. M26927.
GenBank Accession No. M68840.
GenBank Accession No. S64699.
GenBank Accession No. S56903.
GenBank Accession No. U39817.
GenBank Accession No. U49897.
GenBank Accession No. V00497.
GenBank Accession No. V00571.
GenBank Accession No. X01712.
GenBank Accession No. X02747.
GenBank Accession No. X03633.
GenBank Accession No. X13255.
GenBank Accession No. X15149.
GenBank Accession No. X55079.
GenBank Accession No. X55330.
GenBank Accession No. X59798.
GenBank Accession No. Z25884.
GenBank Accession No. Z48804.
GenBank Accession No. AF057310.
Alexandropoulos et al., “v-Fps-responsiveness in the Egr-1 promoter is mediated by serum response elements,”Nucleic Acids Res.,1992, 20(9):2355-2359.
Attar and Gilman, “Expression Cloning of a Novel Zinc Finger Protein That Binds to the c-fos Serum Response Element,”Mol. Cell. Biol.,1992, 12(5):2432-2443.
Bae et al., “Genomic Differences between the Diabetogenic and Nondiabetogenic Variants of Encephalomyocarditis Virus,”Virology,1989, 170:282-287.
Baler et al., “Activation of Human Heat Shock Genes Is Accompanied by Oligomerization, Modification, and Rapid Translocation of Heat Shock Transcription Factor HSF1,”Mol. Cell. Biol.,1993, 13(4):2486-2496.
Bateman et al., “Fusogenic Membrane Glycoproteins As a Novel Class of Genes for the Local and Immune-mediated Control of Tumor Growth,”Cancer Res.,2000, 60:1492-1497.
Bentley et al., “Melanocyte-Specific Expression of the Human Tyrosinase Promoter: Activation by the Microphthalmia Gene Product and Role of the Initiator,”Mol. Cell. Biol.,1994, 14(12):7996-8006.
Berger et al., “Natural and Synthetic Heat Shock Protein Gene Promoters Assayed inDrosophilaCells,”Somatic Cell and Molecular Genetics,1986, 12(5):433-440.
Blackburn et al., “Adenoviral-mediated Transfer of a Heat-inducible Double Suicide Gene into Prostate Carcinoma Cells,”Cancer Res.,1998, 58:1358-1362.
Broach et al., “Vectors for High-Level, Inducible Expression of Cloned Genes in Yeast,”Experimental Manipulation of Gene Expression,Inouye (ed.), 1983, Academic Press, Inc., Orlando, Florida, Chapter 5, pp. 83-117.
Carrol and Taichman, “Characterization of the human involucrin promoter using a transient β-galactosidase assay,”J. Cell Science,1992, 103(4):925-930.
Chaubal et al., “Ep-CAM—A Marker for the Detection of Disseminated Tumor Cells in Patients Suffering from SCCHN,”Anticancer Research,1999, 19(3B):2237-2242.
Cohen et al., “Complete nucleotide sequence of an attenuated hepatitis A virus: Comparison with wild-type virus,”Proc. Natl. Acad. Sci. USA,1987, 84:2497-2501.
Craig, “The Heat Shock Response,”Crit. Rev. Biochem.,1985, 18(3):239-280.
Datta et al., “Ionizing radiation activates transcription of the EGRI gene via CArG elements,”Proc. Natl. Acad. Sci. USA,1992, 89:10149-10153.
Datta et al., “Reactive oxygen intermediates target CC(A/T)6GG sequences to mediate activation of the early growth response 1 transcription factor gene by ionizing radiation,”Proc. Natl. Acad. Sci. USA,1993, 90:2419-2422.
Duechler et al., “Evolutionary relationships within the human rhinovirus genus: Comparison of serotypes 89, 2, and 14,”Proc. Natl. Acad. Sci. USA,1987, 84:2605-2609.
Diaz et al., “Exchange of Viral Promoter/Enhancer Elements with Heterologous Regulatory Sequences Generates Targeted Hybrid Long Terminal Repeat Vectors for Gene Therapy of Melanoma,”J. Virol.,1998, 72:789-795.
Diaz et al., “A lentiviral vector expressing a fusogen

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