Adeno-associated virus-CFTR vector(National Institute of Diabetes & Digestive & Kidney Diseases)

OBJECTIVES:
I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis.
II. Assess the safety of this gene therapy in these patients.
III. Assess the in vivo gene transfer of this vector in these patients.
IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients.
V. Assess the clinical impact of CFTR gene expression following gene transfer in these patients.
VI. Monitor patient immune response directed against CFTR or vector components following vector administration.