Gene Therapy

Gene Genies: One of the most important implications is that we showed the [adenovirus-vector] approach was feasible," asserts Cornell's Ron Crystal. Experiments described in this paper represent two firsts in the burgeoning and fledgling field of gene therapy: the first human trial using a virus-specifically an adenovirus, a microbe isolated from human adenoid tissue-to transport a gene, and the first human trial for cystic fibrosis (CF). CF attacks many organs, but the pulmonary disease that

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"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 ...

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