Development of an AAV CFTR Vector Production System

Our research will develop a scaleable GMP manufacturing process for the production of adeno-associa(AAV) vectors for the treatment of cystic fibrosis. AAV-CFTR vector constructs will be introduced inlines and evaluated for their ability to produce vector particles following addition of requisite panumber of packaging constructs will be assayed in the vector-containing cell lines for their abilityparticle production. Purification of transducing vector particles will be optimized by physical methcentrifugation, and column chromatography. AAV-CFTR vectors will be used in clinical trials for thefibrosis in an attempt to correct the underlying pathophysiology of this disease. Development of thito substantive improvements in treatment of this disease. Development of efficient methods to producvectors could also lead to new products for the treatment of other diseases in addition to cystic fi