Cystic Fibrosis Foundation Therapeutics Inc. made its first major award last month to advance gene editing research in cystic fibrosis. Editas Medicine Inc. will explore the potential of using what some scientists refer to as “a tiny pair of scissors” to snip out the genetic defect that causes CF.
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Cystic fibrosis is a genetic disorder that affects the lungs, pancreas, and other organs. Keep reading to learn how to treat and live with CF.
Every person has two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. A person must inherit two copies of the CFTR gene that contain mutations — one copy from each parent — to have cystic fibrosis.
In an international research project, scientists are examining cystic fibrosis transmembrane conductance regulator (CFTR) mutations to determine which ones cause CF and to provide additional information associated with these mutations. Their findings are available in an online searchable database.
Carrier (or genetic) testing not only plays a key role in the diagnosis of cystic fibrosis, but testing also allows parents to find out what their chances are of having a child with CF to help inform important family planning decisions.
Newborn screening (NBS) is a program run by each state to identify babies born with certain health conditions, including cystic fibrosis. Although a sweat test should ultimately be done to rule out or confirm a CF diagnosis, NBS can help you and your health care providers take immediate steps to keep your child as healthy as possible.