Method of Treating Geographic Atrophy with a Gene Therapy Vector Expressing Soluble CD59

A method of regulating a complement activity disorder in a subject, the method comprising contacting an affected cell of the subject with a pharmaceutical composition comprising a vector carrying a nucleotide sequence encoding a recombinantly engineered human soluble CD59 (sCD59) protein operably linked to a promoter sequence causing expression of the protein in the affected cell, wherein the sCD59 protein comprises at least one mutation resulting in loss of function of glycosylphosphatidylinositol (GPI) anchoring domain resulting in loss of membrane targeting and observing a physiological indicium of the complement activity disorder after the contacting, in comparison to an abnormal amount of the physiological indicium observed prior to the contacting, wherein a decrease after the contacting compared prior to the contacting is a positive indication that the affected cell is treated.

A method of treating a complement disorder comprising contacting a cell with a therapeutically effective amount of a pharmaceutical composition having as an active agent a nucleic acid encoding a human sCD59 protein or a source of expression of a human sCD59 protein comprising administering the pharmaceutical composition to a subject in need thereof.