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Close to 40,000 people in the U.S. have cystic fibrosis, a rare genetic disease. The majority of people with CF are diagnosed by age 2 thanks to newborn screening tests. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make decisions about your health care.
mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.
3 min read
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
6 min read
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.
6 min read