USE OF AAV-EXPRESSED M013 PROTEIN AS AN ANTI-INFLAMMATORY THERAPEUTIC

摘要

Disclosed are methods and compositions for preventing, treating, and/or ameliorating one or more symptoms of inflammation in a mammal. In particular, viral vectors and medicaments containing them are disclosed, which are useful in the prophylaxis, therapy, or amelioration of symptoms of one or more inflammatory-mediated mammalian diseases, such as age-related macular degeneration (AMD), arthritis, Bechet's disease, Best macular dystrophy, corneal inflammation, diabetic retinopathy, drusen formation, dry AMD, dry eye, geographic atrophy, glaucomaocular neovascularization, Lupus erythematosus, macular degeneration, Mallatia Leventinese and Doyne honeycomb retinal dystrophy, nephritis, ocular hypertension, ocular inflammation, recurrent uveitis, Sorsby fundus dystrophy, vasculitis, vitreoretinopathy, wet AMD, or related disorders. In exemplary methods, administration of a pharmaceutical composition comprising a recombinant viral vector that delivers a secretable and cell-penetrating M013 protein or peptide to a subject in need thereof facilitated treatment of particular human disorders such as AMD, ocular neovascularization, uveitis, and related inflammatory ocular disease.

主权项

1. A viral vector comprising:
(a) a polynucleotide that comprises a first isolated nucleic acid segment that encodes a modified, virally-derived, M013 polypeptide; and (b) a second isolated nucleic acid segment encoding a secretion signal peptide; wherein each nucleic acid segment is operably linked to a promoter sequence that expresses the encoded polypeptides in one or more selected mammalian host cells transformed with a viral particle comprising the viral vector.