New treatment for cystic fibrosis Cystic fibrosis, a devastating
illness that clogs a victim’s lungs and renders them susceptible
to infection, is a genetic disease—but one that may see an
unorthodox new treatment in 2002. Sufferers of the disease
possess a damaged version of the CFTR gene, which helps lung
cells to maintain proper fluid balance. Most past attempts at
therapies have involved trying to insert functioning fragments
of CFTR DNA directly into these cells. But these trials have met
with dismal success rates because DNA is notoriously difficult
to introduce into living tissue. The CFTR gene—like all genes
made out of DNA—has to be copied by the cell into RNA before it
is useful. So U.S. researchers took advantage of this basic bit
of biology and tricked CFTR-defective lung cells into accepting
bits of RNA that code for the correct version of the damaged
gene. In quantitative studies, the researchers determined that
16% of all CFTR RNA inside the lung cells came from the foreign
source and was there