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.

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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 affects the inner lining of lung trachea, bronchi, and bronchioles is the most lethal. CF is a hereditary disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR codes for a cell-membrane protein, whose faulty versions cause the respiratory problems characteristic of CF.

"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,"...