Method and compositions for genetic and retinal disease
An inventive method for treatment or prevention of vascular diseases of the retina is provided. A Norrin compound is optionally administered to a subject either directly and/or as expressed by a cell. The presence of the compound is either protective of or therapeutic for a pathological condition of the retina. Preferred pathological conditions are those linked to the absence of or mutation of norrin protein and are preferably Norrie disease, FEVR, or macular degeneration.
1. A process of protecting a retina of a subject from neuronal damage comprising:
administering a Norrin protein in a therapeutically effective amount of 1 to 10 nanograms to an eye to said subject, said Norrin protein binds and stimulates signaling via a frizzled-4 cellular receptor and is selected from the group consisting of: one of SEQ ID Nos. 1-3, an N-terminal truncate fragment thereof, and a mutant thereof, said mutant being a SEQ ID. No. 1 mutation of one of: T27A, S28A, S29A; P36A, R37A, R38A; Y120A, R121A, Y122A; H127A, E129A, E130A or combinations thereof;
said administering of said one of SEQ ID Nos. 1-3, said mutant thereof or said combination thereof protecting the retina of the subject from the neuronal damage.
2. The process of claim 1 wherein said administering is by a route selected from the group consisting of: systemic administration, local administration, injection, topical administration, intraocular, and iontophoretic delivery.
3. The process of claim 1 wherein said subject is selected from the group consisting of: cow, horse, sheep, pig, goat, chicken, cat, dog, mouse, guinea pig, hamster, rabbit, and rat.
4. The process of claim 1 wherein said subject is human.
5. The process of claim 1 wherein said Norrin protein is recombinant.
6. The process of claim 1 wherein said Norrin protein further comprises a marker for monitoring function of the said Norrin protein in the subject to whom said Norrin protein has been administered.
7. The process of claim 1 wherein said marker is selected from the group consisting of: green fluorescent protein, luciferase, and β-galactosidase.
8. The process of claim 1 wherein said Norrin protein is packaged into microspheres.