In 1989 when scientists discovered the defective gene that causes cystic fibrosis, a serious hereditary disorder that primarily strikes children of European descent, it seemed as though a long-hoped-for cure might soon follow. After all, tests in many laboratories showed that providing normal copies of the gene should enable patients to make healthy copies of the protein specified by the gene. If successful, that feat would go a long way toward restoring health in the tens of thousands of people around the world who suffered from cystic fibrosis and typically died in their late 20s
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A Breath of Fresh Air: New Hope for Cystic Fibrosis Treatment (preview)