RNAi modulation of Aha and therapeutic uses thereof

Details RNAi modulation of Aha and therapeutic uses thereof RNAi modulation of Aha and therapeutic uses thereof

2007-05-18

2010-10-12

Wollenberger, Louis (Department: 1635)

Organic compounds -- part of the class 532-570 series

Organic compounds

Carbohydrates or derivatives

Reexamination Certificate

active

07812150

ABSTRACT:
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.

REFERENCES:
patent: 5034506 (1991-07-01), Summerton et al.
patent: 5139941 (1992-08-01), Muzyczka et al.
patent: 5252479 (1993-10-01), Srivastava
patent: 5264221 (1993-11-01), Tagawa et al.
patent: 5328470 (1994-07-01), Nabel et al.
patent: 5602240 (1997-02-01), De Mesmaeker et al.
patent: 5665710 (1997-09-01), Rahman et al.
patent: 5705188 (1998-01-01), Junichi et al.
patent: 6054299 (2000-04-01), Conrad
patent: 6071497 (2000-06-01), Steiner et al.
patent: 6294153 (2001-09-01), Modi
patent: 6344194 (2002-02-01), Sene et al.
patent: 6747014 (2004-06-01), Teng et al.
patent: 6887906 (2005-05-01), Teng et al.
patent: 2005/0255487 (2005-11-01), Khvorova et al.
patent: 2007/0031844 (2007-02-01), Khvorova et al.
patent: 2008/0113931 (2008-05-01), Constien et al.
patent: 101 00 586 (2002-04-01), None
patent: WO 93/24641 (1993-12-01), None
patent: WO 94/13788 (1994-06-01), None
patent: WO 96/40062 (1996-12-01), None
patent: WO 97/04787 (1997-03-01), None
patent: WO 97/30731 (1997-08-01), None
patent: WO 99/32619 (1999-07-01), None
patent: WO 99/53050 (1999-10-01), None
patent: WO 99/61631 (1999-12-01), None
patent: WO 00/22113 (2000-04-01), None
patent: WO 00/22114 (2000-04-01), None
patent: WO 00/44895 (2000-08-01), None
patent: WO 00/50050 (2000-08-01), None
patent: WO 00/66206 (2000-11-01), None
patent: WO 01/60420 (2001-08-01), None
patent: WO 02/053190 (2002-07-01), None
patent: WO 02/066078 (2002-08-01), None
patent: WO 03/067262 (2003-08-01), None
patent: WO 03/070918 (2003-08-01), None
patent: WO 2005/044981 (2005-05-01), None
Vickers et al. (2003) JBC 278:7108-7118.
Holen et al. (2002) Nucleic Acids Res. 30:1757-1766.
PCT International Search Report and Written Opinion, PCT/US2007/069229, Sep. 2, 2008, 9 Pages.
Allen, T.M., and Chonn, A., “Large Unilamellar Liposomes eith Low Uptake into the Reticuloendothelial System,”FEB223: 42-46 (1987).
Anderson, W. F., “Human Gene Therapy,”Nature392:25-30 (1998).
Berkner et al., “Development of Adenovirus Vectors for the Expression of Heterologous Genes,”Biotechniques, 6: 616-629 (1988).
Bucchini et al., “Pancreatic Expression of Human Insulin Gene in Transgenic Mice,”Proc. Natl. Acad. Sci. USA, 83:2511-2515 (1986).
Chen et al., “Gene Therapy for Brain Tumors: Regression of Experimental Gliomas by Adenovirus-Mediated Gene Transfer in vivo,”Proc. Natl. Acad. Sci. USA, 91:3054-3057 (1994).
Cone et al., “High Efficiency Gene Transfer into Mammalian Cells: Generation of Helper-Free Recombinant Retrovirus with Broad Mammalian Host Range,”Proc. Natl. Acad. Sci. USA, 81: 6349-6353 (1984).
Constantinides et al., “Formulation and Intestincal Absorption Enhancement Evaluation of Water-In-Oil Microemulsions Incorporating Medium-Chain Glycerides,”Pharmaceutical Research, 11: 1385-1390 (1994).
Cornetta et al., “Safety Issues Related to Retroviral-Mediated Gene Transfer in Humans,”Human Gene Therapy, 2:5-14 (1991).
Couture et al., “Anti-Gene Therapy: The Use of Ribozymes to Inhibit Gene Function,”TIG, 12: 510-515 (1996).
Dai et al., “HSP90: A Rising Star on the Horizon of Anticancer Targets,”Future Oncol., 4: 529-540 (2005).
Docherty et al., “Nutrient Regulation of Insulin Gene Expression,”FASEB, 8: 20-27 (1994).
Dornburg R., “Reticuloendotheliosis Viruses and Dervied Vectors,”Gene Therapy, 2: 301-310 (1995).
Du Plessis et al., “Topical Delivery of Liposomally Encapsulated Gamma-Interferon,”Antiviral Research, 18: 259-265 (1992).
Eglitis et al., “Retroviral Vecotrs for Introduction of Genes into Mammalian Cells,”Biotechniques, 6: 608-614 (1988).
Fisher et al., “Transduction with Recombinant Adeno-Associated Virus for Gene Therapy is Limited by Leading-Strand Synthesis,”Journal of Virology, 70:520-532 (1996).
Gassmann et al., “Maintenance of an Extrachromosomal Plasmid Vector in Mouse Embryonic Stem Cells,”Proc. Natl. Acad. Sci. USA, 92: 1292-1296 (1995).
Ho et al., “Preparation and Microemulsions Using Polyglycerol Fatty Acid Esters as Surfactant for the Delivery of Protein Drugs,”Journal of Pharmaceutical Sciences, 85: 138-143 (1996).
Hsu et al., “Immunogenicity of Recombinant Adenovirus-Respiratory Syncytial Virus Vaccines with Adenovirs Types 4, 5, and 7 Vectors in Dogs and Chimpanzees,”The Journal of Infectious Diseases, 166: 769-775 (1992).
Hu et al., “Topical Delivery of Cyclosporin A from Non-Ionic Liposomal Systems: An in vivo/in vitro Correlation Study Using Hairless Mouse Skin,”S.T.P. Pharma Science,4: 466-469 (1994).
Lotz et al., “Ahal Binds to the Middle Domain of Hsp90, Contributes to Client Protein Activation, and Stimulated the ATPase Activity of the Molecular Chaperone,”The Journal of Biological Chemistry, 278: 17228-17235 (2003).
Meyer et al., “Structural Basis for Recruitment of the ATPase Activator Aha1 to the Hsp90 Chaperone Machinery,”The EMBO Journal, 23: 511-519 (2004).
Miller A.D., “Retrovirus Packaging Cells,”Human Gene Therapy, 1: 5-14 (1990).
Miyao et al., “Stability and Pharmacokinetic Characteristics of Oligonucleotides Modified at Terminal Linkages in Mice,”Antisense Research and Development, 5: 115-121 (1995).
Muzyczka, N., “Use of Adeno-Associated Virus as a General Transduction Vector for Mammalian Cells,”Current Topics in Microbiology and Immunology, 158: 97-129 (1992).
Panaretou et al., “Activation of the ATPase Activity of Hsp90 by the Stress-Regulated Cochaperone Aha1,”Molecular Cells10: 1307-1318 (2002).
Panaretou et al., “ATP Binding and Hydrolysis are Essential to the Function of the Hsp90 Molecular Chaperone in vivo,”The EMBO Journal, 17: 4829-4836 (1998).
Pearl et al., “Structure, Function, and Mechanism of the Hsp90 Molecular Chaperone,”Advances in Protein Chemistry, 59 157-185 (2002).
Picard D., “Heat-Shock Protein 90, A Chaperone for Folding and Regulation,”Cell. Mol. Life Sci., 59: 1640-1648 (2002).
Pratt et al., “Regulation of Signaling Protein Function and Trafficking by the hsp90/hsp70-Based Chaperone Machinery,”Exp. Biol. Med. 228: 111-133 (2003).
Pratt W.B., “The Role of the hsp90-Based Chaperone System in Signal Transduction by Nuclear Receptors and Receptors Signaling via Map Kinase,”Annu. Rev. Pharmacol. Toxicol., 37: 297-326 (1997).
Prodromou et al., “Structure and Functional Relationships of Hsp90,”Current Cancer Drug Targets, 3: 301-323 (2003).
Prodromou et al., “The ATPase Cycle of Hsp90 Drives a Molecular ‘Clamp’ Via Transient Dimerization of the N-Terminal Domains,”The EMBO Journal, 19:4383-4392 (2000).
Rabinowitz et al., “Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vecotr Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity,”Journal of Virology, 76: 791-801 (2002).
Ritschel W.A., “Microemulsions for Improved Peptide Absorption from the Gastrointestinal Tract,”Meth. Find. Exp. Clin. Phramacol., 13: 205-220 (1991).
Rosenfeld et al., “Adenovirus-Mediated Transfer of a Recombinant #1-Antitrypsin Gene to the Lung Epithelium in Vivo,”Science, 252: 431-434 (1991).
Rosenfeld et al., “In Vi

Affiliated with

Also associated with

Rating

RNAi modulation of Aha and therapeutic uses thereof does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with RNAi modulation of Aha and therapeutic uses thereof, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and RNAi modulation of Aha and therapeutic uses thereof will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-4170781