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Last Updated: April 18, 2024

Claims for Patent: 8,324,182


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Summary for Patent: 8,324,182
Title:Humanized model of membrane attack complex (MAC) formation on murine retina and compositions, kits and methods for treatment of macular degeneration
Abstract: Methods and compositions for treating a subject having age-related macular degeneration (AMD), methods of assaying human macular degeneration (MD), and methods and kits for assaying potential therapeutic agents for treatment of human MD are provided herein.
Inventor(s): Kumar-Singh; Rajendra (Boston, MA), Cashman; Siobhan M (Boston, MA), Ramo; Kasmir (Cambridge, MA)
Assignee: Tufts University (Boston, MA)
Application Number:12/867,566
Patent Claims:1. A method for treating age-related macular degeneration (AMD) in a subject, the method comprising administering a composition into an AMD-affected eye in a subject by ocular injection, wherein said composition comprises a nucleic acid encoding a soluble CD59 protein operably linked to a promoter, wherein said administering results in expression and secretion of said soluble CD59 protein by cells of said AMD-affected eye and said expression results in treatment of AMD-affected tissues or cells in said AMD-affected eye.

2. The method according to claim 1, wherein said nucleic acid is in a viral vector or is a naked nucleic acid.

3. The method according to claim 2, wherein the viral vector is derived from a genetically engineered genome of at least one virus selected from the group consisting of adenovirus, adeno-associated virus, a herpesvirus, and a lentivirus.

4. The method according to claim 3, wherein the lentivirus is a retrovirus.

5. The method according to claim 2, wherein said ocular injection is an intra-ocular injection.

6. The method according to claim 1, wherein the AMD is a dry AMD.

7. The method according to claim 1, wherein said ocular injection is selected from the group consisting of subretinal injection, vitreous injection, intra-ocular injection, subconjunctival injection, and subtenon injection.

8. The method according to claim 1, wherein said ocular injection is an injection of the composition to an external layer of said AMD-affected eye.

9. The method according to claim 1, further comprising administering a therapeutic agent to the AMD-affected eye.

10. The method according to claim 1, wherein the therapeutic agent is selected from the group consisting of: anti-tumor, antiviral, antibacterial, anti-mycobacterial, anti-fungal, anti-proliferative and anti-apoptotic.

11. The method according to claim 10, wherein the therapeutic agent is selected from the group consisting of: a growth factor, an anti-inflammatory agent, a vasopressor agent, a collagenase inhibitor, a steroid, a matrix metalloproteinase inhibitor, an ascorbate, an angiotensin, a calreticulin, a tetracycline, a fibronectin, a collagen, a thrombospondin, a transforming growth factors (TGF), a keratinocyte growth factor (KGF), a fibroblast growth factor (FGF), an insulin-like growth factors (IGFs), an IGF binding protein (IGFBP), an epidermal growth factor (EGF), a platelet derived growth factor (PDGF), a neu differentiation factor (NDF), a hepatocyte growth factor (HGF), a vascular endothelial growth factor (VEGF), a heparin-binding EGF (HBEGF), a thrombospondin, a von Willebrand Factor-C, a heparin, a heparin sulfate, and a hyaluronic acid.

12. The method according to claim 1, where the soluble CD59 protein comprises a deletion of the glycosyl phosphatidyl inositol (GPI) anchoring domain.

13. The method according to claim 1, where the AMD-affected tissues or cells are selected from the group consisting of retina, cornea, vitreous humor, and retinal pigment epithelium.

14. The method according to claim 1, wherein the composition comprises a pharmaceutically acceptable carrier.

15. The method according to claim 1, wherein said soluble CD59 protein spreads extracellularly and affects cells not directly transduced with the nucleic acid of said composition.

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