METHODS FOR TREATING PATIENTS HAVING CFH MUTATIONS WITH CFH-ENCODING VECTORS

摘要

The present disclosure provides methods for treating, preventing, or inhibiting diseases in patients having one or more mutations in complement factor H (CFH), complement component 3 (C3), and complement factor B (CFB) by administering to the patients a recombinant adeno-associated virus (rAAV) vector encoding a CFH polypeptide or biologically active fragment and/or variant thereof.