Organic compounds -- part of the class 532-570 series – Organic compounds – Carbohydrates or derivatives
Reexamination Certificate
2005-07-11
2010-06-29
Ton, Thaian N (Department: 1632)
Organic compounds -- part of the class 532-570 series
Organic compounds
Carbohydrates or derivatives
C435S320100
Reexamination Certificate
active
07745596
ABSTRACT:
To provide a new technique by which efficient transfection is ensured in delivering a target gene into a cell, disclosed is a nucleic acid construct for nuclear import, which comprises a ternary complex consisting of a nucleic acid substance containing a gene to be delivered into the nucleus of a cell, an importin protein (for example, importin-β) capable of passing through the nuclear pore and involved in the nuclear transport, and a binding substance (for example, polyethyleneimine) bound to both of the nucleic acid substance and the importin protein. Nucleic acid transport from outside of a cell into the cell nucleus can be particularly promoted by administering the nucleic acid construct bound to a cell membrane receptor binding factor and/or a membrane fusing substance, or administering the nucleic acid construct encapsulated in a non-viral vector (for example, Sendai virus envelope) having cell membrane permeability and membrane fusing properties.
REFERENCES:
patent: 5824837 (1998-10-01), Chen et al.
patent: 6379966 (2002-04-01), Monahan et al.
O'Neill et al. J Biol Chem 270(39):22701-22704, 1995.
Chan et al. Gene Therapy 7:1690-1697, 2000.
Yang et al. Radiology 228:36-49, 2003.
Tomasoni & Benigni. Current Gene Therapy 4:115-122, 2004.
Gautam et al. Am J Respir Med 1(1):35-46, 2002.
Nagasaki et al. J Controlled Release 103:199-207, Mar. 2005.
Bene, A., et al., Nucleic Acid Res., 32, e142 (2004).
Minari, J., et al., Bull Chem Soc. Jpn. 80, 1091-1098 (2007).
Cho, et al., Polycation Gene Delivery Systems: Escape from Endosomes to Cytosol,Journal of Pharmacy and Pharmacology, JPP 2003, 55:721-734.
Collas, et al., Nuclear Localization Signals: A Driving Force for Nuclear Transport of Plasmid DNA in Zebrafish,Biochem. Cell Biol., 75:633-640 (1997).
Görlich, et al., Distinct Functions for the Two Importin Subunits in Nuclear Protein Import,Nature, vol. 377, 246-248, Sep. 21, 1995.
Hacein-Bey-Abina, et al., LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1,Science, 302, 415 (2003).
Huang, et al., Nonviral Vectors for Gene Therapy,Ernst Wagner, 1999, back cover only.
Imamoto, et al., The Nuclear Pore-Targeting Complex Binds to Nuclear Pores After Association with a Karyophile,FEBS Letters, 368, 415-419 (1995).
Jäkel, et al., Importin β, transportin, RanBP5, and RanBP7 Mediate Nuclear Import of Ribosomal Proteins in Mammalian Cells,The EMBO Journal, vol. 17, No. 15, pp. 4491-4502, 1998.
Kaneda, et al., Hemagglutinating Virus of Japan (HVJ) Envelope Vector as a Versatile Gene Delivery System,Molecular Therapy, vol. 6, No. 2, Aug. 2002.
Kose, et al., Ran-Unassisted Nuclear Migration of a 97-kD Component of Nuclear Pore-Targeting Complex,The Journal of Cell Biology, vol. 139, No. 4, Nov. 17, 1997.
Kutay, et al., Export of Importin α from the Nucleus Is Mediated by a Specific Nuclear Transport Factor,Cell, vol. 90, 1061-1071, Sep. 19, 1997.
Marshall, Gene Therapy Death Prompts Review of Adenovirus Vector,Science, 286, 5448; ProQuest Central p. 2244, Dec. 17, 1999.
Miyata, et al., Block Catiomer Polyplexes with Regulated Densities of Charge and Disulfide Cross-Linking Directed to Enhance Gene Expression,J. Am. Chem. Soc., 126, 2355-2361 (2004).
Nagasaki, et al., Can Nuclear Localization Signals Enhance Nuclear Localization of Plasmid DNA?,Bioconjugate Chem., 14, 282-286 (2003).
Nagoshi, et al., Nuclear Import of Sterol Regulatory Element-Binding Protein-2, a Basic Helix-Loop-Helix-Leucine Zipper (bHLH-Zip)-Containing Transcription Factor, Occurs through the Direct Interaction of Importin β with HLH-Zip,Molecular Biology of the Cell, vol. 10, 2221-2233, Jul. 1999.
Okada, et al., Fusion of Cells by HVJ: Requirement of Concentration of Virus Particles at the Site of Contact of Two Cells for Fusion,Experimental Cell Research, 52, 34-42 (1968).
Oupicky, et al., Stimuli-Responsive Gene Delivery Vectors,Molecular Therapeutics, 5(4):345-350 (2003).
Parente, et al., pH-Dependent Fusion of Phosphatidylcholine Small Vesicles,The Journal of Biological Chemistry, vol. 263, No. 10, pp. 2724-2730, Apr. 5, 1988.
Pollard, et al., A Novel Receptor-Mediated Nuclear Protein Import Pathway,Cell, vol. 86, 985-994, Sep. 20, 1996.
Qian, et al., Targeted Drug Delivery Via the Transferrin Receptor-Mediated Endocytosis Pathway,Pharmacological Reviews, 54:561-587, 2002.
Rexach, et al., Protein Import Into Nuclei: Association and Dissociation Reactions Involving Transport Substrate, Transport Factors, and Nucleoporins,Cell, vol. 83, 683-692, Dec. 1, 1995.
Rolland, Advanced Gene Delivery,CRC Press, 1999, back cover of book only.
Slattum, et al., Efficient in Vitro and in Vivo Expression of Covalently Modified Plasmid DNA,Molecular Therapy, vol. 8, No. 2, Aug. 2003.
Tanimoto, et al., No Enhancement of Nuclear Entry by Direct Conjugation of a Nuclear Localization Signal Peptide to Linearized DNA,Bioconjugate Chem., 14, 1197-1202 (2003).
Vyas, et al., Ligand-Receptor-Mediated Drug Delivery: An Emerging Paradigm in Cellular Drug Targeting,Critical Reviews™ in Therapeutic Drug Carrier Systems, 18(1):1-76 (2001).
Wiethoff, et al., Barriers to Nonviral Gene Delivery,Journal of Pharmaceutical Sciences, vol. 92, No. 2, Feb. 2003.
Yoneda, How Proteins Are Transported from Cytoplasm to the Nucleus,J. Biochem., 121, 811-817 (1997).
Zanta, et al., Gene Delivery: A Single Nuclear Localization Signal Peptide is Sufficient to Carry DNA to the Cell Nucleus,Proc. Natl. Acad. Sci., vol. 96, pp. 91-96, Jan. 1999.
Huang, et al., Nonviral Vectors for Gene Therapy,Ernst Wagner, Chapter 1, pp. 3-22 (1999).
Rolland, Advanced Gene Delivery: from concepts to pharmaceutical products,Harwood Academic, CRC Press, Chapter 4, pp. 65-102 (1999).
Kakimoto Shinji
Kawazu Takeshi
Nagasaki Takeshi
Shinkai Seiji
Fay Sharpe LLP
Japan Science and Technology Agency
Noble Marcia S
Ton Thaian N
LandOfFree
Nuclear transport nucleic acid delivery vector does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Nuclear transport nucleic acid delivery vector, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Nuclear transport nucleic acid delivery vector will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-4162938