Abstract: Compositions, methods and kits are provided for treating complement related disorders in a subject with protein in combination having protein fusions of at least two of a CD46 protein, a CD55 protein and a CD59 protein or with a recombinant chimeric protein having at least two of a CD46 protein, a CD55 protein and a CD59 protein or with nucleic acids encoding these proteins. The composition negatively modulates classical and alternative complement pathways thereby treating complement related disorder such as macular degeneration, age-related macular degeneration, diabetic retinopathy, inflammatory bowel disease, thyroiditis, cryoglobulinaemia, fetal loss, organ graft rejection, cancer, etc.

Abstract: Cell penetrating peptides that target many cells types including cells of the retina and cornea with high efficiency are provided herein. These peptides can be used to deliver cargo molecules across a plasma membrane, without the need for chemical conjugation. Compositions and viral vectors comprising these cell-penetrating peptides are also provided. Methods of using the peptides, compositions and viruses to deliver various agents to target cells and tissues are also provided.

Abstract: Cell penetrating peptides that target many cells types including cells of the retina and cornea with high efficiency are provided herein. These peptides can be used to deliver cargo molecules across a plasma membrane, without the need for chemical conjugation. Compositions and viral vectors comprising these cell-penetrating peptides are also provided. Methods of using the peptides, compositions and viruses to deliver various agents to target cells and tissues are also provided.

Abstract: Compositions, methods and kits are provided for treating complement related disorders in a subject with protein in combination having protein fusions of at least two of a CD46 protein, a CD55 protein and a CD59 protein or with a recombinant chimeric protein having at least two of a CD46 protein, a CD55 protein and a CD59 protein or with nucleic acids encoding these proteins. The composition negatively modulates classical and alternative complement pathways thereby treating complement related disorder such as macular degeneration, age-related macular degeneration, diabetic retinopathy, inflammatory bowel disease, thyroiditis, cryoglobulinaemia, fetal loss, organ graft rejection, cancer, etc.

Abstract: Cell penetrating peptides that target many cells types including cells of the retina and cornea with high efficiency are provided herein. These peptides can be used to deliver cargo molecules across a plasma membrane, without the need for chemical conjugation. Compositions and viral vectors comprising these cell-penetrating peptides are also provided. Methods of using the peptides, compositions and viruses to deliver various agents to target cells and tissues are also provided.

Abstract: Compositions, methods and kits are provided for treating complement related disorders in a subject with protein in combination having protein fusions of at least two of a CD46 protein, a CD55 protein and a CD59 protein or with a recombinant chimeric protein having at least two of a CD46 protein, a CD55 protein and a CD59 protein or with nucleic acids encoding these proteins. The composition negatively modulates classical and alternative complement pathways thereby treating complement related disorder such as macular degeneration, age-related macular degeneration, diabetic retinopathy, inflammatory bowel disease, thyroiditis, cryoglobulinaemia, fetal loss, organ graft rejection, cancer, etc.

Abstract: Methods and kits are provided for inhibiting inflammasome activation in cells of a subject or an inflammation-affected eye in a subject by administering to the subject a composition including a nucleotide sequence encoding a membrane independent CD59 protein operably linked to a promoter for expression and secretion of the membrane independent CD59 protein in the cells or the inflammation-affected eye, the composition inhibiting inflammasome activation.

Abstract: Compositions, methods and kits are provided for treating complement related disorders in a subject with protein in combination having protein fusions of at least two of a CD46 protein, a CD55 protein and a CD59 protein or with a recombinant chimeric protein having at least two of a CD46 protein, a CD55 protein and a CD59 protein or with nucleic acids encoding these proteins. The composition negatively modulates classical and alternative complement pathways thereby treating complement related disorder such as macular degeneration, age-related macular degeneration, diabetic retinopathy, inflammatory bowel disease, thyroiditis, cryoglobulinaemia, fetal loss, organ graft rejection, cancer, etc.

Abstract: Methods, compositions and kits for regulating complement activity or treating a complement activity disorder in a subject using soluble, membrane-independent CD59 protein, methods of assaying human macular degeneration (MD), and methods and kits for assaying potential therapeutic agents for treatment of human MD are provided herein.

Abstract: Methods, compositions and kits for regulating complement activity or treating a complement activity disorder in a subject using soluble, membrane-independent CD59 protein, methods of assaying human macular degeneration (MD), and methods and kits for assaying potential therapeutic agents for treatment of human MD are provided herein.

Abstract: Compositions, methods and kits are provided for treating complement related disorders in a subject with protein in combination having protein fusions of at least two of a CD46 protein, a CD55 protein and a CD59 protein or with a recombinant chimeric protein having at least two of a CD46 protein, a CD55 protein and a CD59 protein or with nucleic acids encoding these proteins. The composition negatively modulates classical and alternative complement pathways thereby treating complement related disorder such as macular degeneration, age-related macular degeneration, diabetic retinopathy, inflammatory bowel disease, thyroiditis, cryoglobulinaemia, fetal loss, organ graft rejection, cancer, etc.

Abstract: Systems, compositions, methods, and kits for identifying potential therapeutic agents for treatment of complement based ocular diseases are provided herein. The methods and kits include a complement component 3 (C3) protein or derivative that is contacted to ocular cells or tissue. Another embodiment of the invention herein provides for diagnosis and/or prognosis of a complement-associated ocular disease. Compositions, methods and kits for regulating or treating a complement-related condition using at least one of CD46 protein, CD55 protein, and a recombinant chimeric soluble terminator of activated complement (STAC) protein or source of the STAC protein. The STAC protein includes an amino acid sequence including at least two of an amino acid sequence of a CD59 protein, an amino acid sequence derived from a CD46 protein, and an amino acid sequence derived from a CD55 protein, optionally further comprising a linker to connect amino acid sequences.

Abstract: A peptide-POD with ability to penetrate and deliver fluorophores, siRNA, DNA and quantum dots to cells in culture and retinal and ocular tissues in vivo is provided herein. POD couples to adenovirus vectors, enhancing tropism for certain cells, potentially providing a safer and more efficacious method to deliver molecules to ocular and other tissues in vivo. POD constructs are therapeutic delivery vehicles for treating cells and tissues, including ocular cells and tissues suffering from retinal 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.

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.

Abstract: A peptide-POD with ability to penetrate and deliver fluorophores, siRNA, DNA and quantum dots to cells in culture and retinal and ocular tissues in vivo is provided herein. POD couples to adenovirus vectors, enhancing tropism for certain cells, potentially providing a safer and more efficacious method to deliver molecules to ocular and other tissues in vivo. POD constructs are therapeutic delivery vehicles for treating cells and tissues, including ocular cells and tissues suffering from retinal degeneration.

Abstract: Adenovirus (Ad) vectors are here provided for treatment of ocular tissues as are suitable methods to transduce photoreceptor (PR) cells, the tissue associated with degeneration. Expression from CMV or chicken beta actin (CBA) promoters in neural retina were compared, and CBA was found to be 173-fold more potent than CMV. Further, the RGD domain in Ad penton was found to play a key role in RPE tropism. Deletion of the RGD domain coupled with the CBA promoter permitted transgene expression in neural retina approximately 667-fold more efficiently than with prior Ad5 vectors. Use of Ad vectors in combination with a 4.7 kb rhodopsin promoter enabled transgene expression exclusively in photoreceptor cells in vivo.

Abstract: The present invention provides improved adenovirus vectors and packaging cell lines. One type of improved adenoviral vector comprises deletions within the E2b region of the adenoviral genome. These E2b-deleted virus are used in conjunction with novel cell lines that constitutively express E2b gene products. The present invention further provides adenoviral vectors deleted for all viral coding regions. These “gutted” vectors permit the transfer of large genes to cells as demonstrated herein by the transfer of the dystrophin gene to the muscle of mice. The E2b-deleted vectors and the gutted vectors provide improved adenoviral vectors useful for a wide variety of gene therapy applications.

Abstract: The present invention provides improved adenovirus vectors and packaging cell lines. One type of improved adenoviral vector comprises deletions within the E2b region of the adenoviral genome. These E2b-deleted virus are used in conjunction with novel cell lines that constitutively express E2b gene products. The present invention further provides adenoviral vectors deleted for all viral coding regions. These "gutted" vectors permit the transfer of large genes to cells as demonstrated herein by the transfer of the dystrophin gene to the muscle of mice. The E2b-deleted vectors and the gutted vectors provide improved adenoviral vectors useful for a wide variety of gene therapy applications.