Modified adeno-associated virus vector capable of expression fro

Chemistry: molecular biology and microbiology – Vector – per se

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536 231, 536 235, 536 241, C12N 15864, C07H 2104

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061657816

ABSTRACT:
Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

REFERENCES:
patent: 4797368 (1989-01-01), Carter et al.
patent: 5587308 (1996-12-01), Carter et al.
patent: 5866696 (1999-02-01), Carter et al.
Verma Inder M. and Somia, Nikunj "Gene Therapy--Promises, Problems and Prospects" Nature 389:239-242, Sep. 18, 1997.
Orkin, Stuart H. and Motulsky, Arno G. "Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy" Dec. 7, 1995.
Marshall, Eliot "Gene Therapy's Growing Pains" Science 269:1050-1055; Aug. 25, 1995.
Kotin, Robert M. "Prospects for the Use of Adeno-Associated Virus as a Vector for Human Gene Therapy" Human Gene Therapy 5:793-801, 1994.
Rosenfeld et al. "Adenovirus-Mediated Transfer of a Recombinant .alpha.1-Antitrypsin Gene to the Lung Epithelium in vivo" Science 252:431-434, Apr. 19, 1991.
Tijssen, P. and Arella, M. "Atlas of Invertebrate Viruses" Chapter 4, pp. 41-53, 1991.
Nabel et al. "Site-Specific Gene Expression in vivo by Direct Gene Transfer into the Arterial Wall" Science 249:1285-1288, Sep. 14, 1990.
Wolff et al. "Direct Gene Transfer into Mouse Muscle in vivo" Science 247:1465-1468, Mar. 23, 1990.
Kaneda et al. "Increased Expression of DNA Cointroduced with Nuclear Protein in Adult Rat-Liver" Science 243:375-378, Jan. 20, 1989.
Mannino, Raphael J. and Gould-Fogerite, Susan "Liposome Mediated Gene Transfer" Biotechniques 6(7):682-690, 1988.
Wu, George Y. and Wu, Catherine H. "Receptor-Mediated Gene Delivery and Expression in vivo" J. Biol. Chem. 263(29):14621-14624, 1986.
Benvensity, Nissim and Reshef, Lea "Direct Introduction of Genes into Rats and Expression of the Genes" Proc. Natl. Acad. Sci. USA 83:9551-9555, Dec. 1986.
Dubensky et al. "Direct Transfection of Viral and Plasmid DNA into the Liver or Spleen of Mice" Proc. Natl. Acad. Sci. USA 81:7529-7533, Dec. 1984.
Seeger et al. "The Cloned Genome of Ground Squirrel Hepatitis Virus is Infectious in the Animal" Proc. Natl. Acad. Sci. USA 81:5849-5852, Sep. 1984.
Nicolau et al. "In Vivo Expression of Rat Insulin after Intravenous Administration of the Liposome-Entrapped Gene for Rat Insulin I" Proc. Natl. Acad. Sci. USA 80:1068-1072, Feb. 1983.
Beaton et al., "Expression From the Adeno-Associated Virus P5 and P19 Promoters is Negatively Regulated in Trans by the Rep Protein," Journal of Virology 63:4450-4454 (1989).
Riordan et al., "Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complimentary DNA," Science 245:1066-1073 (1989).
Collins et al. "Cystic Fibrosis: Molecular Biology and Therapeutic Implications" Science 256:774-779 (1992).
Wickens et al. "Role of the Conserved AAVAAA Sequence: Four AAVAAA Point Mutants Prevent Messenger RNA 3' End Formation," Science 226:1045-1051 (1984).
Susan K. McLaughlin et al. Adeno-Associated Virus General Transduction Vectors: Analysis of Proviral Structures, J. Virol. 62(6):1963-1973 (Jun. 1988).
Srivastava et al. "Construction of a Recombinant Parvovirus B19: Adeno-Associated Virus 2 (AAV) DNA Inverted Terminal repeats are Functional in an AAV-B19 Hybrid Virus," Proc. Natl. Acad. Sci. 86:8078-8082 (Oct. 1989).
Rosenfeld et al., "In Vivo Transfer of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene to the Airway Epithelium," Cell 68:143-155 (Jan. 10, 1992).

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