METHOD AND COMPOSITIONS FOR GENETIC AND RETINAL DISEASE

摘要

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.

主权项

1. A process of protecting a retina of a subject from neuronal damage comprising:
administering a Norrin protein to an eye to said subject, said Norrin protein 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; thereafter the retina developing a pathology or then being exposed to an agent that causes the neuronal damage, said Norrin protein protecting the retina of the subject from the neuronal damage.