"The paper focuses on the use of an adenoviris vector to transfer a normal CFTR cDNA [complementary DNA] to the respiratory epithelium [inner lining] of individuals with CF," explains Ronald G. Crystal, a professor of medicine at the New York Hospital-Cornell University Medical Center. This research started almost three years ago, when Crystal worked as chief of the Pulmonary Branch at the National Heart, Lung, and Blood Institute in Bethesda, Md., and continued when he moved to the Cornell Medical Center.
"The whole concept is that if we could transfer the normal CFTR cDNA to the respiratory epithelium, we could correct the abnormality. In a normal human there are 10 billion airway epithelial cells. Our estimate is that we have to correct about 10 percent of the cells. We performed the first transfer in rodent models [M.A. Rosenfeld et al., 68:143-55, Cell, 1992]."
One of the most important implications of ...
