TREATMENT AND PREVENTION OF NEURODEGENERATIVE DISEASES USING GENE THERAPY

• US 20080292603A1
• Filed: 02/04/2008
• Published: 11/27/2008
• Est. Priority Date: 02/06/2007
• Status: Active Grant

• Current Assignee: Tai June Yoo
• Original Assignee: Tai June Yoo
• Inventors: Yoo, Tai June
• Granted Patent: US 8,247,385 B2
• Time in Patent Office: Days
• US Class Current: 424/93.21
• CPC Class Codes: A61K 2039/53 DNA (RNA) vaccination
• CPC Class Codes: A61K 2039/55522 Cytokines; Lymphokines; Interferons
• CPC Class Codes: A61K 2039/57 characterised by the type of response, e.g. Th1, Th2
• CPC Class Codes: A61K 39/0007 Nervous system antigens; Prions
• CPC Class Codes: A61K 48/00 Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
• CPC Class Codes: A61N 1/327 for enhancing the absorption properties of tissue, e.g. by electroporation
• CPC Class Codes: A61P 25/00 Drugs for disorders of the nervous system
• CPC Class Codes: A61P 25/28 for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
• CPC Class Codes: A61P 29/00 Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
• CPC Class Codes: A61P 37/02 Immunomodulators
• CPC Class Codes: A61P 37/06 Immunosuppressants, e.g. drugs for graft rejection
• CPC Class Codes: A61P 37/08 Antiallergic agents antiasthmatic agents A61P11/06; ophthalmic antiallergics A61P27/14
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: A61P 5/28 Antiandrogens

Abstract

Provided herein are compositions and methods for treating and/or preventing neurodegenerative disease, such as Alzheimer'"'"'s disease. In particular aspects, compositions administered herein encode a cellular immune response element. The compositions may be prepared and administered in such a manner that the cellular immune response element coding sequence is expressed in the subject to which the composition is administered. The compositions include expression systems, delivery systems, and certain cellular immune response element genes.

Claims

1. A method of treating or ameliorating a neurodegenerative disease, said method comprising administering a composition comprising nucleic acid that induces a cellular immune response.

4. The method of claim 1, wherein said nucleic acid encodes a cytokine,

wherein said cytokine comprises one or more selected from the group consisting of IL-4, IL-5, IL-10, IL-13, and TGF-β

or fragments thereof.

11. The method of claim 1, wherein said composition further comprises one or more nucleic acids selected from the group consisting of nucleic acids encoding β

-amyloid, β

-amyloid_1-42, ApoE, ApoE-2, nerve growth factor (NGF), and brain-derived neurotrophic factor (BDNF).

41. The method of claim 1, wherein said nucleic acid is administered to a mammal.

42. The method of claim 41, wherein said mammal is a human.

43. The method of claim 1, wherein said administering comprises one or more methods selected from the group consisting of intravenous injection, intramuscular injection, intraperitoneal injection, subcutaneous injection and electroporation.

44. The method of claim 1, wherein said administering comprises one or methods selected from the group consisting of injection, inhalation, and gene gun.

45. The method of claim 1, wherein said polynucleotide comprises circular DNA comprising an origin or replication (ORI), promoter, and multiple cloning site (MCS).

46. The method of claim 1, wherein said polynucleotide is a plasmid comprising a promoter/enhancer transcriptionally linked to a sequence encoding a cellular immune response element gene.

47. The method of claim 46, wherein said promoter is suitable for expression in eukaryotic cells.

48. The method of claim 1, wherein said nucleic acid is administered with a transfection facilitating material.

50. The method of claim 1, wherein said polynucleotide is delivered by adoptive cellular gene therapy.

51. The method of claim 1, wherein said adoptive gene cellular gene therapy comprises administering a cell capable of targeting delivery of nucleic acid to a site of inflammation.

66. The method of claim 1, wherein said nucleic acid is a vector.

67. The method of claim 66, wherein said vector comprises one or more vectors selected from the group consisted of pVAX1, pUMCV, pGCy, and pLentilox-RES-GFP.

69. The method of claim 1, wherein said composition inhibits or attenuates expression of a gene that increases inflammation.

231. The method of claim 42, wherein said administration results in the decrease of amyloid plaque accumulation in the brain of said mammal.

2-3. -3. (canceled)

5-10. -10. (canceled)

12-16. -16. (canceled)

17. A method of treating or ameliorating a neurodegenerative disease, said method comprising administering a composition comprising a nucleic acid that encodes a polypeptide selected from the group consisting of IL-4, IL-5, IL-10, IL-13, and TGF-β

or fragments thereof.

18. (canceled)

19. A method of treating or ameliorating a neurodegenerative disease, said method comprising administering a composition comprising a nucleic acid that encodes two or more polypeptides selected from the group consisting of IL-4, IL-5, IL-10, IL-13, and TGF-β

or fragments thereof.

24. The method of claim 19, wherein said method further comprises administering nucleic acid that encodes a polypeptide selected from the group consisting of NGF, β

-amyloid, β

-amyloid_1-42, ApoE, and ApoE-2 or fragments thereof.

20-23. -23. (canceled)

25-40. -40. (canceled)

49. (canceled)

52-65. -65. (canceled)

68. (canceled)

70-230. -230. (canceled)